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312<h6 class="h6 highlight-tertiary h8 py-1 ml-0"><span>What is Gene Therapy?</span></h6>
313<h1 class="h1 text-left"><span>Basics of Gene Therapy</span></h1>
314<div class="font-large">�
315Welcome to the heart of genehome, where you will learn about what gene therapy is, as well as the different types of gene therapies that are being studied.</div>
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332<div class="item-text"><h7>What is <br class="gt-content-tile-break"/>gene therapy?</h7></div>
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371<h2 class="h2 pt-5"><span><div itemscope="" itemtype="http://schema.org/Question">
372<span id="what-is-gene-therapy" itemprop="name">What is gene therapy?</span>
373<div class="d-none" itemprop="acceptedAnswer" itemscope="" itemtype="http://schema.org/Answer"> <div itemprop="text">Gene therapy is a type of treatment that uses genetic material with the goal of changing the course of a disease. It is a therapeutic approach that is being investigated for the treatment of multiple diseases. Though many gene therapies are currently in early research or clinical trials 2 gene therapies have already been approved by the US Food and Drug Administration (FDA) as of June 2021.</div></div>
374</div></span></h2>
375<div class="text-septenary">
376<span class="term-dialog"><button aria-expanded="false" class="term-dialog-button" data-template="dialog-194e1f80-9385-427b-a635-ada51ab36bd0">Gene therapy</button><span class="term-dialog-definition" id="dialog-194e1f80-9385-427b-a635-ada51ab36bd0"><span class="term-dialog-title">Gene therapy</span><span class="term-dialog-content">a method of treating genetic diseases at the genetic level (the source) with the goal of changing the course of a disease<br/><br/>
377<a href="/resources/glossary" target="_blank">See glossary for more terms &gt;</a></span></span></span> is a type of treatment that uses genetic material with the goal of changing the course of a disease. It is a therapeutic approach that is being investigated for the treatment of multiple diseases.<sup>1</sup> Though many gene therapies are currently in early research or clinical trials, 2 gene therapies have already been approved by the US Food and Drug Administration (FDA) as of June 2021.<sup>2-4</sup>&nbsp;</div>
378</div>
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384<div class="grid-component container">
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387<h2 class="h2 pt-2"><span><div itemscope="" itemtype="http://schema.org/Question">
388<span itemprop="name">What is the goal of gene therapy?</span>
389<div class="d-none" itemprop="acceptedAnswer" itemscope="" itemtype="http://schema.org/Answer"> <div itemprop="text">The goal of gene therapy is to treat diseases at the genetic level (the source). Gene therapy is a promising treatment option that is being studied for a number of diseases including inherited diseases and cancers.</div></div>
390</div></span></h2>
391<div>
392<p>The goal of gene therapy is to treat diseases at the genetic level (the source). Gene therapy is a treatment method that is being studied for a number of diseases, including inherited diseases and cancers.<sup>1</sup><br/>
393<br/>
394For over 100 years, scientists have studied the human body and the building blocks of DNA. With each mark of progress comes a better understanding of how our bodies work at the genetic level and how we may be able to keep them healthy and free from disease.<sup>5</sup><br/>
395<br/>
396Today, gene therapy continues to build on that strong foundation of DNA research.<sup>1</sup><br/>
397<br/>
398There are 2 major types of gene therapy:<br/>
399<br/>
400<span class="BrandonGrotesqueWeb-Bold">GENE ADDITION</span><br/>
401The addition of genetic materials into the cell to enable the body to produce a functional protein that it could not adequately make before.<sup>6,7,8</sup><br/>
402<br/>
403<span class="BrandonGrotesqueWeb-Bold">GENE EDITING</span><br/>
404The process of directly changing, or editing, a specific site in the genome. The techniques in this therapy include <span class="term-dialog"><a aria-expanded="false" class="term-dialog-button" data-template="dialog-a5c8bf8d-c849-4995-9894-159247148d37">gene correction</a><span class="term-dialog-definition" id="dialog-a5c8bf8d-c849-4995-9894-159247148d37"><span class="term-dialog-title">Gene correction</span><span class="term-dialog-content">a technique that corrects a faulty gene with functional genetic material with the aim of correcting the faulty gene<br/>
405<br/>
406<a href="/resources/glossary" target="_blank">See glossary for more terms &gt;</a></span></span></span>/<span class="term-dialog"><a aria-expanded="false" class="term-dialog-button" data-template="dialog-0918e226-1b44-4e20-87f8-cfa9358cbcfa">insertion</a><span class="term-dialog-definition" id="dialog-0918e226-1b44-4e20-87f8-cfa9358cbcfa"><span class="term-dialog-title">Gene insertion</span><span class="term-dialog-content">a technique that applies genetic material in order to treat a disease at the genetic level<br/>
407<br/>
408<a href="/resources/glossary" target="_blank">See glossary for more terms &gt;</a></span></span></span> and <span class="term-dialog"><a aria-expanded="false" class="term-dialog-button" data-template="dialog-4ea53a76-425e-4dd7-8da3-954b88ef6652">gene inactivation/disruption</a><span class="term-dialog-definition" id="dialog-4ea53a76-425e-4dd7-8da3-954b88ef6652"><span class="term-dialog-title">Gene inactivation</span><span class="term-dialog-content">an approach in gene therapy that turns off or reduces the function of a gene in order to have a therapeutic effect<br/>
409<br/>
410<a href="/resources/glossary" target="_blank">See glossary for more terms &gt;</a></span></span></span>.<sup>6,9</sup></p></div>
411</div>
412</div>
413</div>
414<div class="grid-component container">
415<div class="row">
416<div class="col-md-12">
417<h3 class="h3"><span>Overview of gene addition and gene editing</span></h3>
418<div>
419<table class="d-none d-md-block">
420<tbody>
421<tr>
422<td style="text-align: left; vertical-align: middle; padding: 2px; background-color: #ffffff !important;" width="16%">&nbsp;</td>
423<td class="py-4 px-3" style="border: 1px solid rgb(225, 231, 229); text-align: center; padding: 2px; color:#002643; background-color: #acdff8 !important;">&nbsp;Gene addition</td>
424<td class="pt-4 pb-0" colspan="2" style="border:1px solid #e1e7e5; text-align: center; padding: 2px; background-color: #acdff8 !important; color: #002643" width="28%">&nbsp;Gene editing<br/>
425<br/>
426</td>
427</tr>
428<tr>
429<td class="py-4 px-3" rowspan="2" style="border:1px solid #e1e7e5;text-align: center; vertical-align: top; padding: 2px; background-color: #acdff8 !important;">Mechanism (how it works)</td>
430<td class="py-4 px-3 text-septenary" rowspan="2" style="border:1px solid #e1e7e5;text-align: center; vertical-align: top; padding: 2px; background-color: #ffffff !important;">Inserts functional copies of a gene into target cells using a <span class="term-dialog"><a aria-expanded="false" class="term-dialog-button" data-template="dialog-8f588a74-3f10-46d6-b1f7-2b20681ba5b6">vector</a><span class="term-dialog-definition" id="dialog-8f588a74-3f10-46d6-b1f7-2b20681ba5b6"><span class="term-dialog-title">Vector</span><span class="term-dialog-content">a delivery system used to introduce genetic material into the nucleus<br/>
431<br/>
431
432<a href="/resources/glossary" target="_blank">See glossary for more terms &gt;</a></span></span></span>&nbsp;to overcome the cells' use of a faulty gene<sup>6,7</sup></td>
433<td class="py-3 px-2" style="border:1px solid #e1e7e5;text-align: center; vertical-align: middle; padding: 2px; background-color: #acdff8 !important; color: #002643; " width="28%">&nbsp;Gene inactivation or disruption</td>
434<td class="py-3 px-2" style="border:1px solid #e1e7e5;text-align: center; vertical-align: middle; padding: 2px; background-color: #acdff8 !important; color: #002643; " width="28%">Gene correction or insertion</td>
435</tr>
436<tr>
437<td class="py-4 px-3 text-septenary" style="border:1px solid #e1e7e5;text-align: center; vertical-align: top; padding: 2px; background-color: #ffffff !important;">Creates targeted breaks in DNA <span class="BrandonGrotesqueWeb-Bold">without</span> instructions to repair those breaks, with the aim of disrupting or inactivating the function of a gene<sup>10</sup></td>
438<td class="pt-4 px-3 text-septenary" style="border:1px solid #e1e7e5;text-align: center; vertical-align: top; padding: 2px; background-color: #ffffff !important;">Creates targeted breaks in DNA <span class="BrandonGrotesqueWeb-Bold">with</span> instructions to repair those breaks, with the aim of correcting the function of a gene by inserting functioning genetic material<sup>9</sup></td>
439</tr>
440<tr>
441<td class="py-4 px-3" style="border:1px solid #e1e7e5;text-align: center; vertical-align: top; padding: 2px; background-color: #acdff8 !important;">Key components</td>
442<td class="py-4 px-3 text-septenary" style="border:1px solid #e1e7e5;text-align: center; vertical-align: top; padding: 2px; background-color: #ffffff !important;">Viral vectors containing functional genetic material<sup>6,7</sup></td>
443<td class="pt-4 px-3 text-septenary" colspan="2" style="border:1px solid #e1e7e5;text-align: center; vertical-align: top; padding: 2px; background-color: #ffffff !important;">A targeted editing <span class="term-dialog"><a aria-expanded="false" class="term-dialog-button" data-template="dialog-e80430ac-95fc-4849-8b76-0326464872a6">nuclease</a><span class="term-dialog-definition" id="dialog-e80430ac-95fc-4849-8b76-0326464872a6"><span class="term-dialog-title">Nuclease</span><span class="term-dialog-content">an enzyme that is capable of cleaving the bond between two bases in a nucleic acid at a specific sequence<br/>
444<br/>
445<a href="/resources/glossary" target="_blank">See glossary for more terms &gt;</a></span></span></span>, with or without genetic material to repair DNA breaks<sup>9</sup></td>
446</tr>
447<tr>
448<td class="py-4 px-3" style="border:1px solid #e1e7e5;text-align: center; vertical-align: top; padding: 2px; background-color: #acdff8 !important;">Manufacturing</td>
449<td class="py-4 px-3 text-septenary" style="border:1px solid #e1e7e5;text-align: center; vertical-align: top; padding: 2px; background-color: #ffffff !important;">Therapeutic gene is engineered and packaged into vector for delivery to cells<sup>6,7</sup></td>
450<td class="pt-4 px-3 text-septenary" colspan="2" style="border:1px solid #e1e7e5;text-align: center; vertical-align: top; padding: 2px; background-color: #ffffff !important;">Nuclease and genetic material is engineered and delivered to cells<sup>9</sup></td>
451</tr>
452</tbody>
453</table>
454<table class="d-block d-md-none">
455<tbody>
456<tr>
457<td class="p-3" colspan="2" style="border:1px solid #e1e7e5;text-align: center; vertical-align: middle; padding: 2px; background-color: #acdff8 !important;">Gene Addition</td>
458</tr>
459<tr>
460<td class="p-2" colspan="2" style="border:1px solid #e1e7e5;text-align: center; vertical-align: middle;   font-family: BrandonGrotesqueWeb-medium  padding: 2px; background-color: #5c656d !important; color: #fff" width="28%">Mechanism (how it works)</td>
461</tr>
462<tr>
463<td class="py-4 px-3" colspan="2" style="border:1px solid #e1e7e5;text-align: center; vertical-align: top; padding: 2px; background-color: #ffffff !important;">Inserts functional copies of a gene into target cells using a <span class="term-dialog"><a aria-expanded="false" class="term-dialog-button" data-template="dialog-8f588a74-3f10-46d6-b1f7-2b20681ba5b6">vector</a><span class="term-dialog-definition" id="dialog-8f588a74-3f10-46d6-b1f7-2b20681ba5b6"><span class="term-dialog-title">Vector</span><span class="term-dialog-content">a delivery system used to introduce genetic material into the nucleus<br/>
464<br/>
464
465<a href="/resources/glossary" target="_blank">See glossary for more terms &gt;</a></span></span></span>&nbsp;to overcome the cells' use of a faulty gene<sup>6,7</sup></td>
466</tr>
467<tr>
468<td class="p-2" colspan="2" style="border:1px solid #e1e7e5;text-align: center; vertical-align: middle;   font-family: BrandonGrotesqueWeb-medium  padding: 2px; background-color: #5c656d !important; color: #fff" width="28%">Key Components</td>
469</tr>
470<tr>
471<td class="py-4 px-3" colspan="2" style="border:1px solid #e1e7e5;text-align: center; vertical-align: top; padding: 2px; background-color: #ffffff !important;"><span style="background-color: rgb(255, 255, 255); text-align: center;">Viral vectors containing functional genetic material</span><sup style="text-align: center;">6,7</sup></td>
472</tr>
473<tr>
474<td class="p-2" colspan="2" style="border:1px solid #e1e7e5;text-align: center; vertical-align: middle;   font-family: BrandonGrotesqueWeb-medium  padding: 2px; background-color: #5c656d !important; color: #fff" width="28%">Manufacturing</td>
475</tr>
476<tr>
477<td class="py-4 px-3" colspan="2" style="border:1px solid #e1e7e5;text-align: center; vertical-align: top; padding: 2px; background-color: #ffffff !important;"><span style="background-color: rgb(255, 255, 255); text-align: center;">Therapeutic gene is engineered and packaged into vector for delivery to cells</span><sup style="text-align: center;">6,7</sup></td>
478</tr>
479<tr>
480<td class="p-3" colspan="2" style="border:1px solid #e1e7e5;text-align: center; vertical-align: middle; padding: 2px; background-color: #acdff8 !important;">Gene Editing</td>
481</tr>
482<tr>
483<td class="p-2" colspan="2" style="border:1px solid #e1e7e5;text-align: center; vertical-align: middle;   font-family: BrandonGrotesqueWeb-medium  padding: 2px; background-color: #5c656d !important; color: #fff" width="28%">Mechanism (how it works)</td>
484</tr>
485<tr>
486<td class="p-2" style="border:1px solid #e1e7e5;text-align: center; vertical-align: middle; padding: 2px; background-color: #eaf7fd !important; color: #002643; font-family: BrandonGrotesqueWeb-medium">Gene inactivation or disruption</td>
487<td class="p-2" style="border:1px solid #e1e7e5;text-align: center; vertical-align: middle; padding: 2px; background-color: #eaf7fd !important; color: #002643; font-family: BrandonGrotesqueWeb-medium">Gene correction or insertion</td>
488</tr>
489<tr>
490<td class="py-4 px-3" style="border:1px solid #e1e7e5;text-align: center; vertical-align: top; padding: 2px; background-color: #ffffff !important;"><span style="background-color: rgb(255, 255, 255); text-align: center;">Creates targeted breaks in DNA <span class="BrandonGrotesqueWeb-Bold">without</span> instructions to repair those breaks, with the aim of disrupting or inactivating the function of a gene<sup>10</sup></span></td>
491<td class="py-4 px-3" style="border:1px solid #e1e7e5;text-align: center; vertical-align: top; padding: 2px; background-color: #ffffff !important;"><span style="background-color: rgb(255, 255, 255); text-align: center;">Creates targeted breaks in DNA <span class="BrandonGrotesqueWeb-Bold">with</span> instructions to repair those breaks, with the aim of correcting the function of a gene by inserting functioning genetic material<sup>9</sup></span></td>
492</tr>
493<tr>
494<td class="p-2" colspan="2" style="border:1px solid #e1e7e5;text-align: center; vertical-align: middle;   font-family: BrandonGrotesqueWeb-medium  padding: 2px; background-color: #5c656d !important; color: #fff" width="28%">Key Components</td>
495</tr>
496<tr>
497<td class="py-4 px-3" colspan="2" style="border:1px solid #e1e7e5;text-align: center; vertical-align: top; padding: 2px; background-color: #ffffff !important;"><span style="background-color: rgb(255, 255, 255); text-align: center;">A targeted editing <span class="term-dialog"><a aria-expanded="false" class="term-dialog-button" data-template="dialog-e80430ac-95fc-4849-8b76-0326464872a6">nuclease</a><span class="term-dialog-definition" id="dialog-e80430ac-95fc-4849-8b76-0326464872a6"><span class="term-dialog-title">Nuclease</span><span class="term-dialog-content">
497an enzyme that is capable of cleaving the bond between two bases in a nucleic acid at a specific sequence<br/>
498<br/>
499<a href="/resources/glossary" target="_blank">See glossary for more terms &gt;</a></span></span></span>, with or without genetic material to repair DNA breaks<sup>9</sup></span></td>
500</tr>
501<tr>
502<td class="p-2" colspan="2" style="border:1px solid #e1e7e5;text-align: center; vertical-align: middle;   font-family: BrandonGrotesqueWeb-medium  padding: 2px; background-color: #5c656d !important; color: #fff" width="28%">Manufacturing</td>
503</tr>
504<tr>
505<td class="py-4 px-3" colspan="2" style="border:1px solid #e1e7e5;text-align: center; vertical-align: top; padding: 2px; background-color: #ffffff !important;"><span style="background-color: rgb(255, 255, 255); text-align: center;"><span style="background-color: rgb(255, 255, 255); text-align: center;">Nuclease and genetic material is engineered and delivered to cells</span><sup style="text-align: center;">9</sup></span></td>
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584<h2 class="h2"><span>What are the potential risks of gene therapy?</span></h2>
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586<p>As with any treatment, there are risks associated with gene therapy. Risk depends on the type of gene therapy, type of vector (used to deliver the gene therapy), and the administration method.&nbsp;Some risks can be serious.<sup>11</sup> The safety of gene therapy will continue to be assessed over time.</p>
587<p>Potential risks of gene therapy include:</p></div>
588<div>
589<table class="w-100" style="">
590<tbody>
591<tr>
592<td class="py-4 px-3" style="text-align: left; border: 1px solid #e1e7e5; background-color: #acdff8;">Risk</td>
593<td class="py-4 px-3" style="text-align: left; border: 1px solid #e1e7e5; background-color: #acdff8;">Response</td>
594</tr>
595<tr>
596<td class="py-4 px-3 text-septenary" style="text-align: center; border:1px solid #e1e7e5; text-align: left; vertical-align: top; padding: 2px; background-color: #ffffff;"><span class="BrandonGrotesqueWeb-Bold">Immune reaction</span></td>
597<td class="py-4 px-3 text-septenary" style="text-align: center; border:1px solid #e1e7e5; text-align: left; vertical-align: top; padding: 2px; background-color: #ffffff;">In gene therapy, an immune reaction is an unintentional response from your body. An immune reaction to the gene therapy delivery system or the therapeutic protein could make the therapy less efficient. Antibodies against the gene therapy delivery system can prevent a second dose being administered, if one is needed. Risk of an immune response can be controlled by tailoring the viral dose to the patient.<sup>11</sup></td>
598</tr>
599<tr>
600<td class="py-4 px-3 text-septenary" style="text-align: center; border:1px solid #e1e7e5; text-align: left; vertical-align: top; padding: 2px; background-color: #ffffff;"><span class="BrandonGrotesqueWeb-Bold">Insertional mutagenesis</span></td>
601<td class="py-4 px-3 text-septenary" style="text-align: center; border:1px solid #e1e7e5; text-align: left; vertical-align: top; padding: 2px; background-color: #ffffff;">The consequences of the insertion of DNA in unwanted locations can result in uncontrolled cell division. Insertional mutagenesis is typically associated with gene therapies that integrate into the genome (an example of this is insertional oncogenesis&mdash;healthy cells mutating into cancerous ones). There continues to be a risk of insertional mutagenesis, but every case to date was connected to older technologies that were not yet refined for additional safety. The latest technologies could minimize these risks by not readily integrating into the genome or by using self-inactivating technologies that cannot activate themselves.<sup>11</sup></td>
602</tr>
603<tr>
604<td class="py-4 px-3 text-septenary" style="text-align: center; border:1px solid #e1e7e5; text-align: left; vertical-align: top; padding: 2px; background-color: #ffffff;"><span class="BrandonGrotesqueWeb-Bold">Off-targeting</span></td>
605<td class="py-4 px-3 text-septenary" style="text-align: center; border:1px solid #e1e7e5; text-align: left; vertical-align: top; padding: 2px; background-color: #ffffff;">In the process of gene editing, there is a possibility that the technique used could make changes at a different site than the intended. This can cause changes to healthy genes, which may have unintended effects.<sup>11</sup></td>
606</tr>
607<tr>
608<td class="py-4 px-3 text-septenary" style="text-align: center; border:1px solid #e1e7e5; text-align: left; vertical-align: top; padding: 2px; background-color: #ffffff;"><span class="BrandonGrotesqueWeb-Bold">Unintentional gene inactivation</span></td>
609<td class="py-4 px-3 text-septenary" style="text-align: center; border:1px solid #e1e7e5; text-align: left; vertical-align: top; padding: 2px; background-color: #ffffff;">With any type of gene therapy, there is the risk of unexpected complications that unintentionally prevent the function of another important gene.<sup>11</sup></td>
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658While the goal of gene therapy is to treat people with a given disease or condition, patient safety is a priority. Researchers, clinicians, and the regulatory bodies who review and approve all new gene therapies work together to evaluate the safety and effectiveness of a gene therapy.<br/>
659<br/>
659
660Throughout the gene therapy treatment process, the treating physician and supportive care team provide careful monitoring and management. During the recovery process after gene therapy, they help to manage safety concerns.<br/>
661<br/>
662As with any treatment, patients and caregivers should always discuss their treatment plan and the risks and benefits of a specific gene therapy with their physician.</div>
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722<span itemprop="name">How is a gene therapy developed?</span>
723<div class="d-none" itemprop="acceptedAnswer" itemscope="" itemtype="http://schema.org/Answer">
724<div itemprop="text">To help understand gene therapy, here is a general example of a gene therapy and how it moves from research to a potential reality:
725<ol>
726<li>Scientists discover the genetic basis of a disease, which might be a mutation in a single gene.</li>
727<li>Scientists target their research on ways to modify the specific mutation.</li>
728<li>Different gene therapy techniques are explored in a laboratory in order to figure out which are most effective and show the most promise in treating a genetic disease.</li>
729<li>Once scientists find a technique that they believe has the potential to work, the gene therapy goes through preclinical research and rigorous clinical trials to evaluate the safety and efficacy of the treatment.</li>
730<li>Successful completion of clinical trials may result in approval by the European Medicines Agency (EMA) and/or the US Food and Drug Administration (FDA) and a specific gene therapy becoming available for people with the disease.</li>
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740<p>To help understand gene therapy, here is a general example of a gene therapy and how it moves from research to a potential reality:</p>
741<ol>
742<li>Scientists discover the genetic basis of a disease, which might be a mutation in a single gene.<sup>12</sup></li>
743<li>Scientists target their research on ways to modify the specific mutation.<sup>13</sup></li>
744<li>Different gene therapy techniques are explored in a laboratory in order to figure out which are most effective and show the most promise in treating a genetic disease.<sup>13</sup></li>
745<li>Once scientists find a technique that they believe has the potential to work, the gene therapy goes through preclinical research and rigorous clinical trials to evaluate the safety and&nbsp;<span class="term-dialog"><a aria-expanded="false" class="term-dialog-button" data-template="dialog-07b3b49e-9b01-4bac-8700-c7076c4f72f6">efficacy</a><span class="term-dialog-definition" id="dialog-07b3b49e-9b01-4bac-8700-c7076c4f72f6"><span class="term-dialog-title">Efficacy</span><span class="term-dialog-content">the ability of a medication to achieve its desired effect<br/>
746<br/>
747<a href="/resources/glossary" target="_blank">See glossary for more terms &gt;</a></span></span></span> of the treatment.<sup>14</sup></li>
748<li>Successful completion of clinical trials may result in approval by regulatory authorities such as European Medicines Agency (EMA)&nbsp;and the US Food and Drug Administration (FDA) and a specific gene therapy becoming available for people with the disease.<sup>14,15</sup></li>
749</ol>
750<p>To learn more about the development and history of gene therapy, <a href="/what-is-gene-therapy/history#history">click here</a>.</p></div>
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775<h6 class="h6 h7 bg-desktop-tertiary py-2 pl-3"><span>Other topics you may be interested in:</span></h6>
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777<p><a href="/gene-therapy-process/examples">Examples of Approved Gene Therapies</a></p>
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789<h8>The History and Evolution of gene therapy</h8> </a>
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829<p><span class="BrandonGrotesqueWeb-Bold">References</span></p>
830<p><span class="BrandonGrotesqueWeb-Bold">1.</span> National Institutes of Health. Genetics Home Reference. Help me understand genetics. Accessed July 1, 2021. https://medlineplus.gov/download/genetics/understanding/primer.pdf
831<span class="BrandonGrotesqueWeb-Bold">2.</span> Food and Drug Administration. Approved Cellular and Gene Therapy Products. Accessed July 28, 2021. https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/approved-cellular-and-gene-therapy-products
832<span class="BrandonGrotesqueWeb-Bold">3.</span> Zolgensma (onasemnogene abeparvovec-xio) [prescribing information]. Bannockburn, IL: Kite Novartis, Inc.; 2019.
833<span class="BrandonGrotesqueWeb-Bold">4.</span> Luxturna (voretigene neparvovec-rzyl) [prescribing information]. Philadelphia, PA: Kite Spark Therapeutics, Inc.; 2021.
834<span class="BrandonGrotesqueWeb-Bold">5.</span> Wirth T, Parker N, Yl&auml;-Hertuala. History of gene therapy. <em>Gene.</em> 2013;525(2):162-169.&nbsp;
835<span class="BrandonGrotesqueWeb-Bold">6.</span> FDA Commissioner. What is gene therapy? How does it work? US Food and Drug Administration. Accessed July 1, 2021. https://www.fda.gov/consumers/consumer-updates/what-gene-therapy-how-does-it-work
836<span class="BrandonGrotesqueWeb-Bold">7.</span> Collins M, Thrasher A. Gene therapy: progress and predictions. <em>Proc Biol Sci.</em> 2015;282(1821):20143003.
837<span class="BrandonGrotesqueWeb-Bold">8.</span> STAT Reports. The STAT guide to viral vectors, the linchpin of gene therapy. STAT News; 2019.
838<span class="BrandonGrotesqueWeb-Bold">
8389.</span> Encyclopedia Britannica. Gene editing. Accessed July 1, 2021. https://www.britannica.com/science/gene-editing
839<span class="BrandonGrotesqueWeb-Bold">10.</span> Zhang X, Piedrahita JA. Advances in the generation of transgenic domestic species via somatic cell nuclear transfer. In: Cibelli J, Gurdon J, Wilmut I, et al, Eds. <em>Principles of Cloning.</em> 2nd Edition. Elsevier;2014:95-106.
840<span class="BrandonGrotesqueWeb-Bold">11.</span> Goswami R, Subramanian G, Silayeva L, et al. Gene therapy leaves a vicious cycle. <em>Front Oncol.</em> 2019;9:297
841<span class="BrandonGrotesqueWeb-Bold">12.</span> Amberger JS, Bocchini CA, Scott AF, Hamosh A. OMIM.org: leveraging knowledge across phenotype-gene relationships. <em>Nucleic Acids Res.</em> 2019;47(D1):D1038-D1043.
842<span class="BrandonGrotesqueWeb-Bold">13.</span> Food and Drug Administration. Human gene therapy for rare diseases: guidance for industry. January 2020. Accessed July 1, 2021. https://www.fda.gov/media/113807/download
843<span class="BrandonGrotesqueWeb-Bold">14.</span> Food and Drug Administration. Development &amp; approval process: drugs. Accessed July 1, 2021. https://www.fda.gov/drugs/development-approval-process-drugs
844<span class="BrandonGrotesqueWeb-Bold">15.</span> European Medicines Agency. From laboratory to patient: the journey of a medicine assessed by EMA. Accessed July 1, 2021. https://www.ema.europa.eu/en/documents/other/laboratory-patient-journey-centrally-authorised-medicine_en.pdf</p></div>
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982<div class="modal-header">
983<h5 class="modal-title">
984</h5>
985<button aria-label="Close" class="close" data-dismiss="modal" type="button">
986<span aria-hidden="true"><img alt="Close" src="../images/close-modal.png?v=20260909113039"/></span>
987</button>
988</div>
989<div>
990</div>
991<div class="modal-body">
992<div>
993<div class="col-sm-12">
994<h3>You are about to leave genehome.com</h3>
995<p style="text-align: left;">This link will take you to a website that is outside the control of bluebird bio, Inc. Links are provided for informational purposes only. We do not make or imply any endorsement of external websites.</p>
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997<div class="col-12 col-md-12 pb-0">
998<a "="" class="btn btn-primary mobile-full-width mb-3" data-dismiss="modal" href="##" rel="noopener noreferrer">Cancel</a>
999<a "="" class="ext-link-out btn btn-primary mobile-full-width mb-3" href="##" rel="noopener noreferrer" target="_blank">Continue</a>
1000</div>
1001</div>
1002</div></div>
1003</div>
1004</div>
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1017�
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1022<div class="modal-content">
1023<div class="modal-header">
1024<h5 class="modal-title">
1025</h5>
1026<button aria-label="Close" class="close" data-dismiss="modal" type="button">
1027<span aria-hidden="true"><img alt="Close" src="../images/close-modal.png?v=20260909113039"/></span>
1028</button>
1029</div>
1030<div>
1031</div>
1032<div class="modal-body">
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1034<div class="col-sm-12">
1035<h3>You are about to leave genehome.com</h3>
1036<p style="text-align: left;">This link will take you to the United Kingdom genehome site. By clicking &ldquo;Continue&rdquo; you verify that you are a resident of the United Kingdom.</p>
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1038<div class="col-12 col-md-12 pb-0">
1039<a "="" class="btn btn-primary mobile-full-width mb-3" data-dismiss="modal" href="##" rel="noopener noreferrer">Cancel</a>
1040<a class="ext-link-out btn btn-primary mobile-full-width mb-3" href="https://www.thegenehome.co.uk/" rel="noopener noreferrer" target="_blank">Continue</a>
1041</div>
1042</div>
1043</div></div>
1044</div>
1045</div>
1046</div>
1047</div>
1048<script>�
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1057�
1058�
1059�
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1061<div class="modal leaving-site-style modal-leaving-site-fr fade" data-keyboard="true" id="modal-leaving-site-fr" role="dialog" tabindex="-1">
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1063<div class="modal-content">
1064<div class="modal-header">
1065<h5 class="modal-title">
1066</h5>
1067<button aria-label="Close" class="close" data-dismiss="modal" type="button">
1068<span aria-hidden="true"><img alt="Close" src="../images/close-modal.png?v=20260909113039"/></span>
1069</button>
1070</div>
1071<div>
1072</div>
1073<div class="modal-body">
1074<div>
1075<div class="col-sm-12">
1076<h3>You are about to leave genehome.com</h3>
1077<p style="text-align: left;">This link will take you to the France genehome site. By clicking &ldquo;Continue&rdquo; you verify that you are a resident of France.</p>
1078<div class="row">
1079<div class="col-12 col-md-12 pb-0">
1080<a "="" class="btn btn-primary mobile-full-width mb-3" data-dismiss="modal" href="##" rel="noopener noreferrer">Cancel</a>
1081<a class="ext-link-out btn btn-primary mobile-full-width mb-3" href="https://www.thegenehome.fr/" rel="noopener noreferrer" target="_blank">Continue</a>
1082</div>
1083</div>
1084</div></div>
1085</div>
1086</div>
1087</div>
1088</div>
1089<script>�
1090    function toggleModal(id) {�
1091        var element = $("#" + id);�
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1098�
1099�
1100�
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1104<div class="modal-content">
1105<div class="modal-header">
1106<h5 class="modal-title">
1107</h5>
1108<button aria-label="Close" class="close" data-dismiss="modal" type="button">
1109<span aria-hidden="true"><img alt="Close" src="../images/close-modal.png?v=20260909113039"/></span>
1110</button>
1111</div>
1112<div>
1113</div>
1114<div class="modal-body">
1115<div>
1116<div class="col-sm-12">
1117<h3>You are about to leave genehome.com</h3>
1118<p style="text-align: left;">This link will take you to the European Union genehome site. By clicking &ldquo;Continue&rdquo; you verify that you are a resident of the European Union.</p>
1119<div class="row">
1120<div class="col-12 col-md-12 pb-0">
1121<a "="" class="btn btn-primary mobile-full-width mb-3" data-dismiss="modal" href="##" rel="noopener noreferrer">Cancel</a>
1122<a class="ext-link-out btn btn-primary mobile-full-width mb-3" href="https://www.thegenehome.eu/" rel="noopener noreferrer" target="_blank">Continue</a>
1123</div>
1124</div>
1125</div></div>
1126</div>
1127</div>
1128</div>
1129</div>
1130<script>�
1131    function toggleModal(id) {�
1132        var element = $("#" + id);�
1133        if (element.hasClass('in')) {�
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1139�
1140�
1141�
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1146<div class="modal-header">
1147<h5 class="modal-title">
1148</h5>
1149<button aria-label="Close" class="close" data-dismiss="modal" type="button">
1150<span aria-hidden="true"><img alt="Close" src="../images/close-modal.png?v=20260909113039"/></span>
1151</button>
1152</div>
1153<div>
1154</div>
1155<div class="modal-body">
1156<div>
1157<h3>There's more!</h3>
1158<p style="text-align: left;">Start receiving the latest info on gene therapy today.&nbsp;</p>
1159<a class="delayed-cta-link" href="/sign-up" rel="noopener noreferrer" target="_blank">Stay informed &gt;</a></div>
1160</div>
1161</div>
1162</div>
1163</div>
1164<script>�
1165    function toggleModal(id) {�
1166        var element = $("#" + id);�
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1173�
1174�
1175�
1176</script>
vendor: 2 bytes, line 1176
1176
1177<script id="Cookiebot" src="https://consent.cookiebot.com/uc.js" data-cbid="19a0fad5-01e2-4794-9855-ae3d4724d796" type="text/javascript" async></script>
1177
1178<div class="modal leaving-site-style modal-leaving-site-de fade" data-keyboard="true" id="modal-leaving-site-de" role="dialog" tabindex="-1">
1179<div class="modal-dialog modal-dialog-centered" role="document">
1180<div class="modal-content">
1181<div class="modal-header">
1182<h5 class="modal-title">
1183</h5>
1184<button aria-label="Close" class="close" data-dismiss="modal" type="button">
1185<span aria-hidden="true"><img alt="Close" src="../images/close-modal.png?v=20260909113039"/></span>
1186</button>
1187</div>
1188<div>
1189</div>
1190<div class="modal-body">
1191<div>
1192<div class="col-sm-12">
1193<h3>You are about to leave genehome.com</h3>
1194<p style="text-align: left;">This link will take you to the Germany genehome site. By clicking &ldquo;Continue&rdquo; you verify that you are a resident of Germany</p>
1195<div class="row">
1196<div class="col-12 col-md-12 pb-0">
1197<a "="" class="btn btn-primary mobile-full-width mb-3" data-dismiss="modal" href="##" rel="noopener noreferrer">Cancel</a>
1198<a class="ext-link-out btn btn-primary mobile-full-width mb-3" href="https://www.thegenehome.de" rel="noopener noreferrer" target="_blank">Continue</a>
1199</div>
1200</div>
1201</div></div>
1202</div>
1203</div>
1204</div>
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1216�
1217�
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1218
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vendor: 2 bytes, line 1222
1222
1223<script src="../scripts/build/app.min.js?v=20260909113039"></script>
vendor: 2 bytes, line 1223
1223
1224<script src="../scripts/build/PageNavigation.js?v=20260909113039"></script>
vendor: 2 bytes, line 1224
1224
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1225
1226</body>
1227</html>

Line numbers count LF bytes from the start of the resource, as the search results do. Vendor segments are library code the classifier recognised; they are stored but not indexed. Bytes are shown as Latin1 characters, one per byte.