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<span>Genes and Gene Therapy</span>
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Genes and Gene Therapy
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<h3>Basics</h3>
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<a title="Go to: Start Here" href="#cat_51">Start Here</a>
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<h3>Learn More</h3>
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<a title="Go to: Related Issues" href="#cat_47">Related Issues</a>
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<h3>Research</h3>
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<a title="Go to: Clinical Trials" href="#cat_27">Clinical Trials</a>
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<h3>For You</h3>
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<a title="Go to: Children" href="#cat_8">Children</a>
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<h2>Summary</h2>
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<div id="topic-summary" class="syndicate"><p>Genes are the building blocks of inheritance. Passed from parent to child, they contain
instructions for making proteins. If genes don't produce the right proteins or don't produce them
correctly, a child can have a <a href="https://medlineplus.gov/geneticdisorders.html">genetic disorder</a>. </p>
<p>Gene therapy is an experimental technique that uses genes to treat or prevent disease.
The most common form of gene therapy involves inserting a normal gene to replace an
abnormal gene. Other approaches include:</p><ul>
<li> Swapping an abnormal gene for a normal one</li>
<li> Repairing an abnormal gene</li>
<li> Altering the degree to which a gene is turned on or off</li>
</ul>
<p>Although there is much hope for gene therapy, it is still experimental. </p></div>
</section>
<a name="cat_51"></a>
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<h2>Start Here
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<li>
<a href="https://www.mayoclinic.org/tests-procedures/gene-therapy/about/pac-20384619?p=1" target="TheNewWin" data-sid="62598" >Gene Therapy</a>
<span class="desc-text">
<span class="orgs"> (Mayo Foundation for Medical Education and Research)</span>
</span>
<span class="also-lang"> Also in <a target="TheNewWin2" href="https://www.mayoclinic.org/es/tests-procedures/gene-therapy/about/pac-20384619?p=1" data-sid="130519">Spanish</a> </span>
</li>
<li>
<a href="https://www.ashg.org/discover-genetics-old/genetics-basics/" target="TheNewWin" data-sid="88189" >Genetics Basics: Six Things You Should Know</a>
<span class="desc-text">
<span class="orgs"> (American Society of Human Genetics)</span>
</span>
</li>
<li>
<a href="https://www.fda.gov/consumers/consumer-updates/how-gene-therapy-can-cure-or-treat-diseases" target="TheNewWin" data-sid="156166" >How Gene Therapy Can Cure or Treat Diseases</a>
<span class="desc-text">
<span class="orgs"> (Food and Drug Administration)</span>
</span>
<span class="also-lang"> Also in <a target="TheNewWin2" href="https://www.fda.gov/consumers/articulos-para-el-consumidor-en-espanol/como-la-terapia-genica-puede-curar-o-tratar-enfermedades" data-sid="156246">Spanish</a> </span>
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<a href="https://www.nhlbi.nih.gov/health/genetic-therapies" target="TheNewWin" data-sid="155372" data-org="NHLBI">What Are Genetic Therapies?</a>
<span class="desc-text">
<img width="25" height="16" alt="From the National Institutes of Health" title="From the National Institutes of Health" src="https://medlineplus.gov/images/nih.png" class="imgdesc">
<span class="orgs"> (National Heart, Lung, and Blood Institute)</span>
</span>
<span class="also-lang"> Also in <a target="TheNewWin2" href="https://www.nhlbi.nih.gov/es/salud/terapias-geneticas" data-sid="156088">Spanish</a> </span>
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<a href="https://www.genome.gov/about-genomics/fact-sheets/A-Brief-Guide-to-Genomics" target="TheNewWin" data-sid="110698" data-org="NHGRI">Brief Guide to Genomics: DNA, Genes and Genomes</a>
<span class="desc-text">
<img width="25" height="16" alt="From the National Institutes of Health" title="From the National Institutes of Health" src="https://medlineplus.gov/images/nih.png" class="imgdesc">
<span class="orgs"> (National Human Genome Research Institute)</span>
</span>
<span class="also-lang"> Also in <a target="TheNewWin2" href="https://www.genome.gov/es/about-genomics/fact-sheets/Breve-guia-de-genomica" data-sid="120190">Spanish</a> </span>
</li>
<li>
<a href="https://www.genome.gov/about-genomics/fact-sheets/Epigenomics-Fact-Sheet" target="TheNewWin" data-sid="78223" data-org="NHGRI">Epigenomics</a>
<span class="desc-text">
<img width="25" height="16" alt="From the National Institutes of Health" title="From the National Institutes of Health" src="https://medlineplus.gov/images/nih.png" class="imgdesc">
<span class="orgs"> (National Human Genome Research Institute)</span>
</span>
<span class="also-lang"> Also in <a target="TheNewWin2" href="https://www.genome.gov/es/about-genomics/fact-sheets/Epigenomica" data-sid="120189">Spanish</a> </span>
</li>
<li>
<a href="https://www.genome.gov/FAQ/Pharmacogenomics" target="TheNewWin" data-sid="78106" data-org="NHGRI">Frequently Asked Questions about Pharmacogenomics</a>
<span class="desc-text">
<img width="25" height="16" alt="From the National Institutes of Health" title="From the National Institutes of Health" src="https://medlineplus.gov/images/nih.png" class="imgdesc">
<span class="orgs"> (National Human Genome Research Institute)</span>
</span>
<span class="also-lang"> Also in <a target="TheNewWin2" href="https://www.genome.gov/es/FAQ/Preguntas-frecuentes-sobre-la-farmacogenomica" data-sid="144346">Spanish</a> </span>
</li>
<li>
<a href="https://www.genome.gov/FAQ/Genetics-Disease-Prevention-and-Treatment" target="TheNewWin" data-sid="88190" data-org="NHGRI">Genetics, Disease Prevention and Treatment</a>
<span class="desc-text">
<img width="25" height="16" alt="From the National Institutes of Health" title="From the National Institutes of Health" src="https://medlineplus.gov/images/nih.png" class="imgdesc">
<span class="orgs"> (National Human Genome Research Institute)</span>
</span>
</li>
<li>
<a href="https://medlineplus.gov/genetics/" data-sid="40583" data-org="NLM">Genetics: MedlinePlus Genetics</a>
<span class="desc-text">
<img width="25" height="16" alt="From the National Institutes of Health" title="From the National Institutes of Health" src="https://medlineplus.gov/images/nih.png" class="imgdesc">
<span class="orgs"> (National Library of Medicine)</span>
</span>
<span class="also-lang"> Also in <a href="https://medlineplus.gov/spanish/genetica/" data-sid="156089">Spanish</a> </span>
</li>
<li>
<a href="https://medlineplus.gov/genetics/understanding/testing/sequencing" data-sid="107068" data-org="NLM">What are whole exome sequencing and whole genome sequencing?: MedlinePlus Genetics</a>
<span class="desc-text">
<img width="25" height="16" alt="From the National Institutes of Health" title="From the National Institutes of Health" src="https://medlineplus.gov/images/nih.png" class="imgdesc">
<span class="orgs"> (National Library of Medicine)</span>
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<span class="also-lang"> Also in <a href="https://medlineplus.gov/spanish/genetica/entender/pruebas/secuenciacion/" data-sid="156090">Spanish</a> </span>
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<li>
<a href="https://medlineplus.gov/genetics/understanding/howgeneswork/epigenome" data-sid="107063" data-org="NLM">What is epigenetics?: MedlinePlus Genetics</a>
<span class="desc-text">
<img width="25" height="16" alt="From the National Institutes of Health" title="From the National Institutes of Health" src="https://medlineplus.gov/images/nih.png" class="imgdesc">
<span class="orgs"> (National Library of Medicine)</span>
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<span class="also-lang"> Also in <a href="https://medlineplus.gov/spanish/genetica/entender/comofuncionangenes/epigenetica/" data-sid="156091">Spanish</a> </span>
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<a href="https://medlineplus.gov/genetics/understanding/dtcgenetictesting/ancestrytesting" data-sid="107066" data-org="NLM">What is genetic ancestry testing?: MedlinePlus Genetics</a>
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<img width="25" height="16" alt="From the National Institutes of Health" title="From the National Institutes of Health" src="https://medlineplus.gov/images/nih.png" class="imgdesc">
<span class="orgs"> (National Library of Medicine)</span>
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<span class="also-lang"> Also in <a href="https://medlineplus.gov/spanish/genetica/entender/pruebasdirectasalconsumidor/pruebasdeascendencia/" data-sid="156247">Spanish</a> </span>
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<a href="https://biobeat.nigms.nih.gov/2023/08/what-is-pharmacology/" target="TheNewWin" data-sid="62279" data-org="NIGMS">What is Pharmacology?</a>
<span class="desc-text">
<img width="25" height="16" alt="From the National Institutes of Health" title="From the National Institutes of Health" src="https://medlineplus.gov/images/nih.png" class="imgdesc">
<span class="orgs"> (National Institute of General Medical Sciences)</span>
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<h2>Specifics
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<a href="https://patienteducation.asgct.org/disease-treatments" target="TheNewWin" data-sid="97369" >Disease Treatments</a>
<span class="desc-text">
<span class="orgs"> (American Society of Gene &amp; Cell Therapy)</span>
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<section id="cat_79_section">
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<h2>Statistics and Research
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<li>
<a href="https://www.genome.gov/human-genome-project" target="TheNewWin" data-sid="9896" data-org="NHGRI">All about the Human Genome Project (HGP)</a>
<span class="desc-text">
<img width="25" height="16" alt="From the National Institutes of Health" title="From the National Institutes of Health" src="https://medlineplus.gov/images/nih.png" class="imgdesc">
<span class="orgs"> (National Human Genome Research Institute)</span>
</span>
</li>
<li>
<a href="https://www.nih.gov/news-events/news-releases/coming-age-gene-therapy-review-past-path-forward" target="TheNewWin" data-sid="139288" data-org="NIH">Coming of Age of Gene Therapy: A Review of the Past and Path Forward</a>
<span class="desc-text">
<img width="25" height="16" alt="From the National Institutes of Health" title="From the National Institutes of Health" src="https://medlineplus.gov/images/nih.png" class="imgdesc">
<span class="orgs"> (National Institutes of Health)</span>
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<li>
<a href="https://www.nih.gov/news-events/news-releases/nih-researchers-create-new-viral-vector-improved-gene-therapy-sickle-cell-disease" target="TheNewWin" data-sid="139287" data-org="NHLBI">NIH Researchers Create New Viral Vector for Improved Gene Therapy in Sickle Cell Disease</a>
<span class="desc-text">
<img width="25" height="16" alt="From the National Institutes of Health" title="From the National Institutes of Health" src="https://medlineplus.gov/images/nih.png" class="imgdesc">
<span class="orgs"> (National Heart, Lung, and Blood Institute)</span>
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<section id="cat_27_section">
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<h2>Clinical Trials
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<li>
<a href="https://clinicaltrials.gov/search?intr=%22gene+therapy%22&amp;aggFilters=status:not%20rec" target="TheNewWin" data-sid="28545" data-org="NIH">ClinicalTrials.gov: Gene Therapy</a>
<span class="desc-text">
<img width="25" height="16" alt="From the National Institutes of Health" title="From the National Institutes of Health" src="https://medlineplus.gov/images/nih.png" class="imgdesc">
<span class="orgs"> (National Institutes of Health)</span>
</span>
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<h2>Journal Articles
<span class="subheading">References and abstracts from MEDLINE/PubMed (National Library of Medicine)</span>
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<li>
<a href="https://www.ncbi.nlm.nih.gov/pubmed/39747998" target="TheNewWin" data-sid="24069" >Article: Immunologic investigations into transgene directed immune-mediated myositis following delandistrogene moxeparvovec gene...</a>
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</span>
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<li>
<a href="https://www.ncbi.nlm.nih.gov/pubmed/39424303" target="TheNewWin" data-sid="24069" >Article: Clinical development and proof of principle testing of new regenerative vascular...</a>
<span class="desc-text">
</span>
</li>
<li>
<a href="https://www.ncbi.nlm.nih.gov/pubmed/39256350" target="TheNewWin" data-sid="24069" >Article: Interim safety and efficacy of gene therapy for RLBP1-associated retinal dystrophy:...</a>
<span class="desc-text">
</span>
</li>
<li>
<a href="https://pubmed.ncbi.nlm.nih.gov/?term=%22Genetic+Therapy%22%5Bmajr%3Anoexp%5D+OR+%22Genes%22%5Bmajr%3Anoexp%5D+AND+humans%5Bmh%5D+AND+english%5Bla%5D+AND+%22last+1+Year%22+%5Bedat%5D+AND+%28patient+education+handout%5Bpt%5D+OR+guideline%5Bpt%5D+OR+clinical+trial%5Bpt%5D%29++NOT+%28letter%5Bpt%5D+OR+case+reports%5Bpt%5D+OR+editorial%5Bpt%5D+OR+comment%5Bpt%5D%29+AND+free+full+text%5Bsb%5D+" target="TheNewWin" data-sid="24069" >Genes and Gene Therapy -- see more articles</a>
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<h2>Reference Desk
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<li>
<a href="https://www.genome.gov/about-genomics/fact-sheets/Biological-Pathways-Fact-Sheet" target="TheNewWin" data-sid="129807" data-org="NHGRI">Biological Pathways</a>
<span class="desc-text">
<img width="25" height="16" alt="From the National Institutes of Health" title="From the National Institutes of Health" src="https://medlineplus.gov/images/nih.png" class="imgdesc">
<span class="orgs"> (National Human Genome Research Institute)</span>
</span>
<span class="also-lang"> Also in <a target="TheNewWin2" href="https://www.genome.gov/es/about-genomics/fact-sheets/Vias-Biologicas" data-sid="129808">Spanish</a> </span>
</li>
<li>
<a href="https://www.genome.gov/about-genomics/fact-sheets/Chromosomes-Fact-Sheet" target="TheNewWin" data-sid="129815" data-org="NHGRI">Chromosomes</a>
<span class="desc-text">
<img width="25" height="16" alt="From the National Institutes of Health" title="From the National Institutes of Health" src="https://medlineplus.gov/images/nih.png" class="imgdesc">
<span class="orgs"> (National Human Genome Research Institute)</span>
</span>
<span class="also-lang"> Also in <a target="TheNewWin2" href="https://www.genome.gov/es/about-genomics/fact-sheets/Cromosomas" data-sid="129814">Spanish</a> </span>
</li>
<li>
<a href="https://www.genome.gov/about-genomics/fact-sheets/DNA-Microarray-Technology" target="TheNewWin" data-sid="129809" data-org="NHGRI">DNA Microarray Technology</a>
<span class="desc-text">
<img width="25" height="16" alt="From the National Institutes of Health" title="From the National Institutes of Health" src="https://medlineplus.gov/images/nih.png" class="imgdesc">
<span class="orgs"> (National Human Genome Research Institute)</span>
</span>
<span class="also-lang"> Also in <a target="TheNewWin2" href="https://www.genome.gov/es/about-genomics/fact-sheets/Tecnologia-de-micromatriz-de-ADN" data-sid="129810">Spanish</a> </span>
</li>
<li>
<a href="https://www.ncbi.nlm.nih.gov/gtr/" target="TheNewWin" data-sid="25600" data-org="NCBI">Genetic Testing Registry</a>
<span class="desc-text">
<img width="25" height="16" alt="From the National Institutes of Health" title="From the National Institutes of Health" src="https://medlineplus.gov/images/nih.png" class="imgdesc">
<span class="orgs"> (National Center for Biotechnology Information)</span>
</span>
</li>
<li>
<a href="https://www.genome.gov/genetics-glossary" target="TheNewWin" data-sid="77753" data-org="NHGRI">Talking Glossary of Genetic Terms</a>
<span class="desc-text">
<img width="25" height="16" alt="From the National Institutes of Health" title="From the National Institutes of Health" src="https://medlineplus.gov/images/nih.png" class="imgdesc">
<span class="orgs"> (National Human Genome Research Institute)</span>
</span>
<span class="also-lang"> Also in <a target="TheNewWin2" href="https://www.genome.gov/es/genetics-glossary" data-sid="39176">Spanish</a> </span>
</li>
<li>
<a href="https://www.genome.gov/about-genomics/fact-sheets/Transcriptome-Fact-Sheet" target="TheNewWin" data-sid="129811" data-org="NHGRI">Transcriptome</a>
<span class="desc-text">
<img width="25" height="16" alt="From the National Institutes of Health" title="From the National Institutes of Health" src="https://medlineplus.gov/images/nih.png" class="imgdesc">
<span class="orgs"> (National Human Genome Research Institute)</span>
</span>
<span class="also-lang"> Also in <a target="TheNewWin2" href="https://www.genome.gov/es/about-genomics/fact-sheets/Transcriptoma" data-sid="129812">Spanish</a> </span>
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<h2>Find an Expert
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<li>
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<h2>Children
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<a href="https://kidshealth.org/en/parents/about-genetics.html" target="TheNewWin" data-sid="114242" >All about Genetics (For Parents)</a>
<span class="desc-text">
<span class="orgs"> (Nemours Foundation)</span>
</span>
<span class="also-lang"> Also in <a target="TheNewWin2" href="https://kidshealth.org/es/parents/about-genetics.html" data-sid="117639">Spanish</a> </span>
</li>
<li>
<a href="https://kidshealth.org/en/parents/gene-mutations.html" target="TheNewWin" data-sid="150286" >Gene Changes (Mutations)</a>
<span class="desc-text">
<span class="orgs"> (Nemours Foundation)</span>
</span>
<span class="also-lang"> Also in <a target="TheNewWin2" href="https://kidshealth.org/es/parents/gene-mutations.html" data-sid="152176">Spanish</a> </span>
</li>
<li>
<a href="https://kidshealth.org/en/kids/what-is-gene.html" target="TheNewWin" data-sid="24129" >What Is a Gene?</a>
<span class="desc-text">
<span class="orgs"> (Nemours Foundation)</span>
</span>
<span class="also-lang"> Also in <a target="TheNewWin2" href="https://kidshealth.org/es/kids/what-is-gene.html" data-sid="118409">Spanish</a> </span>
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<h2>Teenagers
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<a href="https://kidshealth.org/en/teens/genes-genetic-disorders.html" target="TheNewWin" data-sid="24131" >Basics on Genes and Genetic Disorders</a>
<span class="desc-text">
<span class="orgs"> (Nemours Foundation)</span>
</span>
<span class="also-lang"> Also in <a target="TheNewWin2" href="https://kidshealth.org/es/teens/genes-genetic-disorders.html" data-sid="72106">Spanish</a> </span>
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<h2>MEDICAL ENCYCLOPEDIA</h2>
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<li><a data-sid="82428" href="https://medlineplus.gov/ency/article/002371.htm">Genes</a></li>
<li><a data-sid="82288" href="https://medlineplus.gov/ency/article/002048.htm">Genetics</a></li>
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<h2>National Institutes of Health</h2>
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The primary NIH organization for research on
<span class="italic">Genes and Gene Therapy</span> is the
<a href="http://www.genome.gov/" target="TheNewWin">National Human Genome Research Institute</a>
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<li><a data-sid="155579" href="https://magazine.medlineplus.gov/article/gene-therapies-are-a-promising-path-to-treating-rare-diseases" target="_blank">Gene Therapies are a Promising Path to Treating Rare Diseases</a></li>
<li><a data-sid="158206" href="https://magazine.medlineplus.gov/article/tesha-samuels-advocates-for-others-after-living-with-sickle-cell-disease" target="_blank">Tesha Samuels Advocates for Others After Living with Sickle Cell Disease</a></li>
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