{"id":12693,"date":"2025-09-13T13:29:00","date_gmt":"2025-09-13T13:29:00","guid":{"rendered":"https:\/\/www.nextias.com\/blog\/?p=12693"},"modified":"2025-09-15T12:18:32","modified_gmt":"2025-09-15T12:18:32","slug":"gene-therapy","status":"publish","type":"post","link":"https:\/\/www.nextias.com\/blog\/gene-therapy\/","title":{"rendered":"Gene Therapy: Types, Advantages, Disadvantages &amp; More"},"content":{"rendered":"\n<p>Gene therapy is a medical technique that treats or prevents diseases by inserting functional genes to correct genetic defects. It offers potential cures for hereditary disorders, cancer, and chronic conditions. Despite its promise, challenges like immunity and ethics remain. This article aims to provide about Gene Therapy and Its Types, Advantages, Disadvantages &amp; More.<\/p><div id=\"ez-toc-container\" class=\"ez-toc-v2_0_56_1 counter-hierarchy ez-toc-counter ez-toc-transparent ez-toc-container-direction\">\n<div class=\"ez-toc-title-container\">\n<span class=\"ez-toc-title-toggle\"><\/span><\/div>\n<nav><ul class='ez-toc-list ez-toc-list-level-1 ' ><li class='ez-toc-page-1 ez-toc-heading-level-2'><a class=\"ez-toc-link ez-toc-heading-1\" href=\"https:\/\/www.nextias.com\/blog\/gene-therapy\/#What_is_Gene_Therapy\" title=\"What is Gene Therapy?\">What is Gene Therapy?<\/a><\/li><li class='ez-toc-page-1 ez-toc-heading-level-2'><a class=\"ez-toc-link ez-toc-heading-2\" href=\"https:\/\/www.nextias.com\/blog\/gene-therapy\/#Types_of_Gene_Therapy\" title=\"Types of Gene Therapy\">Types of Gene Therapy<\/a><ul class='ez-toc-list-level-3'><li class='ez-toc-heading-level-3'><a class=\"ez-toc-link ez-toc-heading-3\" href=\"https:\/\/www.nextias.com\/blog\/gene-therapy\/#Germ_Line_Gene_Therapy\" title=\"Germ Line Gene Therapy\">Germ Line Gene Therapy<\/a><\/li><li class='ez-toc-page-1 ez-toc-heading-level-3'><a class=\"ez-toc-link ez-toc-heading-4\" href=\"https:\/\/www.nextias.com\/blog\/gene-therapy\/#Somatic_Gene_Therapy\" title=\"Somatic Gene Therapy\">Somatic Gene Therapy<\/a><\/li><\/ul><\/li><li class='ez-toc-page-1 ez-toc-heading-level-2'><a class=\"ez-toc-link ez-toc-heading-5\" href=\"https:\/\/www.nextias.com\/blog\/gene-therapy\/#Advantages\" title=\"Advantages \">Advantages <\/a><\/li><li class='ez-toc-page-1 ez-toc-heading-level-2'><a class=\"ez-toc-link ez-toc-heading-6\" href=\"https:\/\/www.nextias.com\/blog\/gene-therapy\/#Disadvantages\" title=\"Disadvantages \">Disadvantages <\/a><\/li><li class='ez-toc-page-1 ez-toc-heading-level-2'><a class=\"ez-toc-link ez-toc-heading-7\" href=\"https:\/\/www.nextias.com\/blog\/gene-therapy\/#Way_Forward\" title=\"Way Forward\">Way Forward<\/a><\/li><li class='ez-toc-page-1 ez-toc-heading-level-2'><a class=\"ez-toc-link ez-toc-heading-8\" href=\"https:\/\/www.nextias.com\/blog\/gene-therapy\/#Latest_Developments_in_Gene_Therapy\" title=\"Latest Developments in Gene Therapy\">Latest Developments in Gene Therapy<\/a><\/li><li class='ez-toc-page-1 ez-toc-heading-level-2'><a class=\"ez-toc-link ez-toc-heading-9\" href=\"https:\/\/www.nextias.com\/blog\/gene-therapy\/#Conclusion\" title=\"Conclusion\">Conclusion<\/a><\/li><li class='ez-toc-page-1 ez-toc-heading-level-2'><a class=\"ez-toc-link ez-toc-heading-10\" href=\"https:\/\/www.nextias.com\/blog\/gene-therapy\/#Frequently_Asked_Questions_FAQs\" title=\"Frequently Asked Questions (FAQs)\">Frequently Asked Questions (FAQs)<\/a><ul class='ez-toc-list-level-3'><li class='ez-toc-heading-level-3'><a class=\"ez-toc-link ez-toc-heading-11\" href=\"https:\/\/www.nextias.com\/blog\/gene-therapy\/#Which_is_the_first_gene_therapy_in_India\" title=\"Which is the first gene therapy in India?\">Which is the first gene therapy in India?<\/a><\/li><li class='ez-toc-page-1 ez-toc-heading-level-3'><a class=\"ez-toc-link ez-toc-heading-12\" href=\"https:\/\/www.nextias.com\/blog\/gene-therapy\/#Which_disease_was_first_successfully_cured_by_gene_therapy\" title=\"Which disease was first successfully cured by gene therapy?\">Which disease was first successfully cured by gene therapy?<\/a><\/li><li class='ez-toc-page-1 ez-toc-heading-level-3'><a class=\"ez-toc-link ez-toc-heading-13\" href=\"https:\/\/www.nextias.com\/blog\/gene-therapy\/#Who_founded_gene_therapy\" title=\"Who founded gene therapy?\">Who founded gene therapy?<\/a><\/li><li class='ez-toc-page-1 ez-toc-heading-level-3'><a class=\"ez-toc-link ez-toc-heading-14\" href=\"https:\/\/www.nextias.com\/blog\/gene-therapy\/#What_was_the_first_gene_therapy_drug\" title=\"What was the first gene therapy drug?\">What was the first gene therapy drug?<\/a><\/li><li class='ez-toc-page-1 ez-toc-heading-level-3'><a class=\"ez-toc-link ez-toc-heading-15\" href=\"https:\/\/www.nextias.com\/blog\/gene-therapy\/#What_is_gene_therapy_according_to_NCERT\" title=\"What is gene therapy according to NCERT?\">What is gene therapy according to NCERT?<\/a><\/li><\/ul><\/li><\/ul><\/nav><\/div>\n\n\n\n\n<h2 class=\"wp-block-heading has-text-color has-link-color wp-elements-2ee9bc28208da99851395451ddc96c93\" style=\"color:#015aa7\"><span class=\"ez-toc-section\" id=\"What_is_Gene_Therapy\"><\/span><strong>What is Gene Therapy?<\/strong><span class=\"ez-toc-section-end\"><\/span><\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li>It is an attempt to correct Hereditary Disease in any person. It is a collection of methods that allows correction of a gene defect that has been diagnosed in a child\/embryo. Here genes are inserted into a person\u2019s cells and tissues to treat a disease. Correction of a genetic defect involves delivery of a normal gene into the individual or embryo to take over the function of and compensate for the non-functional gene.<\/li>\n<\/ul>\n\n\n<div class=\"wp-block-image\">\n<figure class=\"aligncenter size-full is-resized\"><img loading=\"lazy\" decoding=\"async\" width=\"485\" height=\"283\" src=\"https:\/\/wp-images.nextias.com\/cdn-cgi\/image\/format=auto\/blog\/uploads\/2024\/10\/gene-augmentation-therapy.png\" alt=\"gene augmentation therapy\" class=\"wp-image-12694\" style=\"width:485px;height:auto\" srcset=\"https:\/\/wp-images.nextias.com\/cdn-cgi\/image\/format=auto\/blog\/uploads\/2024\/10\/gene-augmentation-therapy.png 485w, https:\/\/wp-images.nextias.com\/cdn-cgi\/image\/format=auto\/blog\/uploads\/2024\/10\/gene-augmentation-therapy-442x258.png 442w, https:\/\/wp-images.nextias.com\/cdn-cgi\/image\/format=auto\/blog\/uploads\/2024\/10\/gene-augmentation-therapy-150x88.png 150w\" sizes=\"auto, (max-width: 485px) 100vw, 485px\" \/><figcaption class=\"wp-element-caption\">Figure: Gene Augmentation Therapy<\/figcaption><\/figure>\n<\/div>\n\n\n<h2 class=\"wp-block-heading has-text-color has-link-color wp-elements-e247330bc7fd2e581bf063f58e6edaa3\" style=\"color:#015aa7\"><span class=\"ez-toc-section\" id=\"Types_of_Gene_Therapy\"><\/span><strong>Types of Gene Therapy<\/strong><span class=\"ez-toc-section-end\"><\/span><\/h2>\n\n\n\n<h3 class=\"wp-block-heading has-text-color has-link-color wp-elements-bcd11aa2752b4c4054590475d814f8fe\" style=\"color:#ff6a00\"><span class=\"ez-toc-section\" id=\"Germ_Line_Gene_Therapy\"><\/span><strong>Germ Line Gene Therapy<\/strong><span class=\"ez-toc-section-end\"><\/span><\/h3>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Here <strong>germ cells (sperm or egg)<\/strong> are modified by the introduction of functional genes and are integrated in the genome. Therefore, changes due to therapy would be heritable and would be passed on to the later generation. Theoretically, this approach should be highly effective in counteracting <strong>genetic disease and hereditary disorders,<\/strong> but at present there are various technical difficulties and ethical concerns that make it difficult to try in human beings.<\/li>\n<\/ul>\n\n\n\n<h3 class=\"wp-block-heading has-text-color has-link-color wp-elements-2db9caae479764a5a825d92a2acd7a72\" style=\"color:#ff6a00\"><span class=\"ez-toc-section\" id=\"Somatic_Gene_Therapy\"><\/span><strong>Somatic Gene Therapy<\/strong><span class=\"ez-toc-section-end\"><\/span><\/h3>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Here therapeutic genes are transferred into the somatic cells of a patient. Any modifications and effects will be restricted to the individual patient only and will not be inherited by the patients offspring or any later generation.<\/li>\n<\/ul>\n\n\n\n<h2 class=\"wp-block-heading has-text-color has-link-color wp-elements-6c333cc6e9087f8047974c361c7df5df\" style=\"color:#015aa7\"><span class=\"ez-toc-section\" id=\"Advantages\"><\/span><strong>Advantages <\/strong><span class=\"ez-toc-section-end\"><\/span><\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li>To give someone who is born with a genetic disease or who develops cancer a chance of the normal life.<\/li>\n\n\n\n<li>Gene silencing is a concept that in itself is self-efficient for management of many diseases.<\/li>\n\n\n\n<li>Gene therapy has the potential to eliminate and prevent hereditary diseases, such as <strong>cystic fibrosis, and is a possible cure for heart disease, AIDS and cancer.<\/strong><\/li>\n<\/ul>\n\n\n\n<h2 class=\"wp-block-heading has-text-color has-link-color wp-elements-fcd5fbc9bfd2a8a4b44991ad82cae0e0\" style=\"color:#015aa7\"><span class=\"ez-toc-section\" id=\"Disadvantages\"><\/span><strong>Disadvantages<\/strong> <span class=\"ez-toc-section-end\"><\/span><\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>Short-lived Nature of Gene Therapy:<\/strong> For gene therapy to be accepted as a permanent cure for any disorder, it has to be ensured that the therapeutic DNA introduced into the target cells undergoes expression for long and the cells expressing the therapeutic DNA are stable and long-lived.<\/li>\n\n\n\n<li><strong>Immune Response: <\/strong>As long as a foreign entity is introduced into human tissues, evolutionary events have predisposed the immune system to mount an attack against the invader. There is always a risk of awakening the immune system in such a way as to counteract gene therapy.<\/li>\n\n\n\n<li><strong>Multigenic Disorders:<\/strong> Diseases caused by variation in a single gene are theoretically the best candidates for gene therapy. Unfortunately, the most prevalent disorders, such as <strong>heart disease, hypertension, Alzheimer&#8217;s, arthritis, and diabetes, <\/strong>emerge from variations in many genes working in combination.<\/li>\n\n\n\n<li><strong>Ethical Considerations:<\/strong> Some ethical aspects of gene therapy lead to questions such as deciding what is normal and what is not; deciding whether disabilities are diseases and whether they should be cured; and whether the search for a cure demeans the lives of people who have disabilities.<\/li>\n<\/ul>\n\n\n\n<h2 class=\"wp-block-heading has-text-color has-link-color wp-elements-1e603a2ea607b35340e5267a97dffca5\" style=\"color:#015aa7\"><span class=\"ez-toc-section\" id=\"Way_Forward\"><\/span><strong>Way Forward<\/strong><span class=\"ez-toc-section-end\"><\/span><\/h2>\n\n\n\n<p>This exciting new method has just started to make news. One day this word will be part of our daily homes. Making a transition out from what it is today in the field of medicine can be done using it. As scientists continue to discover genes and their purposes, the avenues of treatment are endless. Our genome is our body&#8217;s blueprint. In some respects, the key to our future lies locked away in our genome. One&#8217;s life will be forever changed as researchers begin to unlock that blueprint. We know in some sense that our fate is really in the genes.<\/p>\n\n\n\n<h2 class=\"wp-block-heading has-text-color has-link-color wp-elements-4f4a1a2e4c6d11036625319d77df5e44\" style=\"color:#015aa7\"><span class=\"ez-toc-section\" id=\"Latest_Developments_in_Gene_Therapy\"><\/span><strong>Latest Developments in Gene Therapy<\/strong><span class=\"ez-toc-section-end\"><\/span><\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Recent developments show promise in furthering the directions of precision medicine and genetic disease treatments. Perhaps<strong> CRISPR-based treatments <\/strong>are now moving towards an in vivo kind to treat genetic diseases like sickle cell disease<strong> (SCD)<\/strong> directly in the body, enhancing accessibility and decreasing dependence on chemotherapy\u200b.<\/li>\n\n\n\n<li>Simultaneously, new clinical studies investigate gene therapy for hereditary diseases such as amyloidosis, with LNPs used to deliver genes effectively to the exact tissue. This indicates single-dose therapies that would be enough to correct the faulty gene.<\/li>\n\n\n\n<li>Going forward in research highlights these innovations morphing away from conventional therapies into targeted genetic therapies that promise personalized efficient treatments for chronic illnesses, cancers, and genetic disorders.<\/li>\n<\/ul>\n\n\n\n<h2 class=\"wp-block-heading has-text-color has-link-color wp-elements-d0adc4bd9eb2e9b9b187dc32f0e06642\" style=\"color:#015aa7\"><span class=\"ez-toc-section\" id=\"Conclusion\"><\/span><strong>Conclusion<\/strong><span class=\"ez-toc-section-end\"><\/span><\/h2>\n\n\n\n<p>It offers long-term solutions for genetic disorders, cancer, and chronic diseases by correcting faulty genes at their source. With advancements like <strong>CRISPR<\/strong>, its potential grows, but challenges such as immune responses, high costs, and ethical concerns remain. As research advances, gene therapy is poised to become a key element of precision medicine, improving patient outcomes and quality of life.<\/p>\n\n\n\n<p class=\"has-background\" style=\"background-color:#ebecf0\"><strong>Further Reading:<\/strong> <a href=\"https:\/\/www.nextias.com\/blog\/crispr-cas9\/\" data-type=\"link\" data-id=\"https:\/\/www.nextias.com\/blog\/crispr-cas9\/\"><strong>CRISPR-Cas9<\/strong><\/a><\/p>\n\n\n\n<h2 class=\"wp-block-heading has-text-color has-link-color wp-elements-618812723183593a2c916a1fd5800128\" style=\"color:#015aa7\"><span class=\"ez-toc-section\" id=\"Frequently_Asked_Questions_FAQs\"><\/span><strong>Frequently Asked Questions (FAQs)<\/strong><span class=\"ez-toc-section-end\"><\/span><\/h2>\n\n\n<div id=\"rank-math-faq\" class=\"rank-math-block\">\n<div class=\"rank-math-list \">\n<div id=\"faq-question-1729776050946\" class=\"rank-math-list-item\">\n<h3 class=\"rank-math-question \"><span class=\"ez-toc-section\" id=\"Which_is_the_first_gene_therapy_in_India\"><\/span><strong>Which is the first gene therapy in India?<\/strong><span class=\"ez-toc-section-end\"><\/span><\/h3>\n<div class=\"rank-math-answer \">\n\n<p>India&#8217;s first indigenous gene therapy, <strong>CAR-T cell therapy<\/strong>, was launched on April 4, 2024, at the Indian Institute of Technology (IIT) Bombay.<\/p>\n\n<\/div>\n<\/div>\n<div id=\"faq-question-1729776067923\" class=\"rank-math-list-item\">\n<h3 class=\"rank-math-question \"><span class=\"ez-toc-section\" id=\"Which_disease_was_first_successfully_cured_by_gene_therapy\"><\/span><strong>Which disease was first successfully cured by gene therapy?<\/strong><span class=\"ez-toc-section-end\"><\/span><\/h3>\n<div class=\"rank-math-answer \">\n\n<p>The first disease successfully treated with it was <strong>severe combined immunodeficiency (SCID).<\/strong><\/p>\n\n<\/div>\n<\/div>\n<div id=\"faq-question-1729776084280\" class=\"rank-math-list-item\">\n<h3 class=\"rank-math-question \"><span class=\"ez-toc-section\" id=\"Who_founded_gene_therapy\"><\/span><strong>Who founded gene therapy?<\/strong><span class=\"ez-toc-section-end\"><\/span><\/h3>\n<div class=\"rank-math-answer \">\n\n<p>The concept of gene therapy was first proposed by scientists W. French Anderson, Michael Blaese, and Kenneth Culver in the 1980s. W. French Anderson is often regarded as the &#8220;father of gene therapy&#8221; for leading the first approved human gene therapy trial in 1990, which treated a four-year-old girl with severe<strong> combined immunodeficiency (SCID)<\/strong>.<\/p>\n\n<\/div>\n<\/div>\n<div id=\"faq-question-1729776101769\" class=\"rank-math-list-item\">\n<h3 class=\"rank-math-question \"><span class=\"ez-toc-section\" id=\"What_was_the_first_gene_therapy_drug\"><\/span><strong>What was the first gene therapy drug?<\/strong><span class=\"ez-toc-section-end\"><\/span><\/h3>\n<div class=\"rank-math-answer \">\n\n<p>The first approved gene therapy drug was <strong>Gendicine<\/strong>, developed by Shenzhen SiBiono GenTech. It was approved in <strong>2003<\/strong> by China\u2019s regulatory authorities to treat<strong> head and neck squamous cell carcinoma<\/strong>. Gendicine uses an adenovirus to deliver the <strong>p53 tumor suppressor gene<\/strong> into cancer cells, helping to inhibit their growth.<\/p>\n\n<\/div>\n<\/div>\n<div id=\"faq-question-1729776150904\" class=\"rank-math-list-item\">\n<h3 class=\"rank-math-question \"><span class=\"ez-toc-section\" id=\"What_is_gene_therapy_according_to_NCERT\"><\/span><strong>What is gene therapy according to NCERT?<\/strong><span class=\"ez-toc-section-end\"><\/span><\/h3>\n<div class=\"rank-math-answer \">\n\n<p>According to NCERT, gene therapy is a treatment that involves inserting genes into a person&#8217;s cells and tissues to treat diseases, especially hereditary diseases.<\/p>\n\n<\/div>\n<\/div>\n<\/div>\n<\/div>","protected":false},"excerpt":{"rendered":"<p>Gene therapy is a medical technique that treats or prevents diseases by inserting functional genes to correct genetic defects.<\/p>\n","protected":false},"author":9,"featured_media":12724,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[63],"tags":[72],"class_list":["post-12693","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-science-and-technology","tag-gs-3"],"_links":{"self":[{"href":"https:\/\/www.nextias.com\/blog\/wp-json\/wp\/v2\/posts\/12693","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.nextias.com\/blog\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.nextias.com\/blog\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.nextias.com\/blog\/wp-json\/wp\/v2\/users\/9"}],"replies":[{"embeddable":true,"href":"https:\/\/www.nextias.com\/blog\/wp-json\/wp\/v2\/comments?post=12693"}],"version-history":[{"count":7,"href":"https:\/\/www.nextias.com\/blog\/wp-json\/wp\/v2\/posts\/12693\/revisions"}],"predecessor-version":[{"id":21850,"href":"https:\/\/www.nextias.com\/blog\/wp-json\/wp\/v2\/posts\/12693\/revisions\/21850"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.nextias.com\/blog\/wp-json\/wp\/v2\/media\/12724"}],"wp:attachment":[{"href":"https:\/\/www.nextias.com\/blog\/wp-json\/wp\/v2\/media?parent=12693"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.nextias.com\/blog\/wp-json\/wp\/v2\/categories?post=12693"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.nextias.com\/blog\/wp-json\/wp\/v2\/tags?post=12693"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}