What Is Gene Therapy? (for Parents)

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href="/es/parents/gene-therapy.html">¿Qué es la terapia génica?</a> </div> <div> <div id="reviewerBoxTop"> <div id="reviewedByTop"> Medically reviewed by: <a href="https://www.nemours.org/about/kidshealth-medical-editors.html" target="_blank">Larissa Hirsch, MD</a> </div> </div> </div> <div> <div id="departmentSpecialty"> <a href="https://www.nemours.org/services/pediatric-genetics.html" class="rev-multi" target="_blank">Learn About Genetics at Nemours Children's Health</a> </div> </div> </div> <div id="khcontent"> <div id="khcontent_article" class="khLeft"> <span class="ISI_MESSAGE"></span> <ul id="msArticleControls"> <li class="ac-readspeaker"> <div class="rs_skip rs_skip_en rsbtn_kidshealth rs_preserve" id="readspeaker_button1"> <a onclick="rsUsed('1')" rel="nofollow" class="rsbtn_play rsen" title="Listen to this page using ReadSpeaker" href="//app-na.readspeaker.com/cgi-bin/rsent?customerid=5202&lang=en_us&readid=khcontent_article&url=kidshealth.org/en/parents/gene-therapy.html" 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They also determine things like the likelihood of getting some kinds of diseases.</p> <p>Scientists believe that every human has about 20,000 to 25,000 genes. If even one of these genes is missing or has <a href="/en/parents/gene-mutations.html">changed (mutated)</a>, it can cause a disease. Sometimes a mutation is inherited, just like curly hair or brown eyes. Others happen for no known reason, without having been passed on by a parent.</p> <h3>What Is Gene Therapy?</h3> <p>Gene therapy involves using genes to fight or prevent diseases. It might mean replacing a gene that isn’t working properly, adding a "good" gene into a person who has a disease, or blocking a gene that is causing a problem.</p> <p>Gene therapy is already available to treat some diseases. Many other types of gene therapy are being studied to see how safe they are, and if they can work to treat or prevent other diseases. This process is slow because experts have to make sure that the benefits are worth the possible risks.</p> <p>With time, gene therapy has the potential to prevent and treat many diseases.</p> </div> </div> </div> <div id="reviewerBox"> <div id="reviewedBy"><strong>Medically reviewed by: </strong><a href="https://www.nemours.org/about/kidshealth-medical-editors.html" class="vcard-link" target="_blank">Larissa Hirsch, MD</a> </div> <div id="reviewedDate">Date reviewed: February 2024</div> </div> <span class="ISI_MESSAGE">  </span> </div> <div id="khcontent_article_aside" class="khRight"> <div> <aside aria-labelledby="moreOnThisTopicHeader"> <div id="kh-mott-box"> <h2 id="moreOnThisTopicHeader" class="JSMott">More on this topic for:</h2> <ul id="kh-mott-tabs" role="tablist" class="JSMott"> <li data-section="parents" role="presentation"><button type="button" role="tab" aria-controls="parents_panel" id="parents_tab" tabindex="0" aria-selected="true" class="kh-mott-active">Parents</button></li> </ul> <div id="parents_panel" role="tabpanel" aria-labelledby="parents_tab"> <h2 class="sansJSMott">More on this topic<br/>For Parents</h2> <ul id="kh-mott-parents-links" class="kh-mott-links" style="display: block"> <li> <a href="/en/parents/genetics.html"> Genetic Testing </a> </li> <li> <a href="/en/parents/gene-mutations.html"> Gene Changes (Mutations) </a> </li> <li> <a href="/en/parents/genetic-counseling.html"> Genetic Counseling </a> </li> <li> <a href="/en/parents/about-epigenetics.html"> Epigenetics </a> </li> <li> <a href="/en/parents/center/about-sickle-cell-disease.html"> Sickle Cell Disease (Topic Center) </a> </li> <li> <a href="/en/parents/baby-has-birth-defect.html"> If Your Baby Has a Birth Defect </a> </li> </ul> </div> <button id="kh-mott-toggle" data-expanded="false">View more</button> </div> </aside> </div> <aside aria-label="Nemours Message"> <div id="kh-partner-ad" class="rs_skip rs_preserve"> <div class="kh-inline"><iframe title="Partner Message" id="mobad_in_if" src="/content/kidshealth/us/misc/inline_ad.html" style="display: block; 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What Is Gene Therapy? (for Parents) | Nemours KidsHealth