{"id":352,"date":"2015-01-08T07:01:15","date_gmt":"2015-01-08T07:01:15","guid":{"rendered":"https:\/\/delveinsightblog.wordpress.com\/?p=352"},"modified":"2024-09-17T15:27:25","modified_gmt":"2024-09-17T09:57:25","slug":"a-crispr-cas9-approach-for-gene-therapy-the-hot-new-rd-field","status":"publish","type":"post","link":"https:\/\/www.delveinsight.com\/blog\/a-crispr-cas9-approach-for-gene-therapy-the-hot-new-rd-field","title":{"rendered":"Revolutionizing Gene Editing: CRISPR-Cas9 Paves the Way for Cutting-Edge Gene Therapy Innovations!"},"content":{"rendered":"<div id=\"ez-toc-container\" class=\"ez-toc-v2_0_76 counter-hierarchy ez-toc-counter ez-toc-white ez-toc-container-direction\">\n<p class=\"ez-toc-title\" style=\"cursor:inherit\">Table of Contents<\/p>\n<label for=\"ez-toc-cssicon-toggle-item-69f3fbc8ad062\" class=\"ez-toc-cssicon-toggle-label\"><span class=\"\"><span class=\"eztoc-hide\" style=\"display:none;\">Toggle<\/span><span class=\"ez-toc-icon-toggle-span\"><svg style=\"fill: #999;color:#999\" xmlns=\"http:\/\/www.w3.org\/2000\/svg\" class=\"list-377408\" width=\"20px\" height=\"20px\" viewBox=\"0 0 24 24\" fill=\"none\"><path d=\"M6 6H4v2h2V6zm14 0H8v2h12V6zM4 11h2v2H4v-2zm16 0H8v2h12v-2zM4 16h2v2H4v-2zm16 0H8v2h12v-2z\" fill=\"currentColor\"><\/path><\/svg><svg style=\"fill: #999;color:#999\" class=\"arrow-unsorted-368013\" xmlns=\"http:\/\/www.w3.org\/2000\/svg\" width=\"10px\" height=\"10px\" viewBox=\"0 0 24 24\" version=\"1.2\" baseProfile=\"tiny\"><path d=\"M18.2 9.3l-6.2-6.3-6.2 6.3c-.2.2-.3.4-.3.7s.1.5.3.7c.2.2.4.3.7.3h11c.3 0 .5-.1.7-.3.2-.2.3-.5.3-.7s-.1-.5-.3-.7zM5.8 14.7l6.2 6.3 6.2-6.3c.2-.2.3-.5.3-.7s-.1-.5-.3-.7c-.2-.2-.4-.3-.7-.3h-11c-.3 0-.5.1-.7.3-.2.2-.3.5-.3.7s.1.5.3.7z\"\/><\/svg><\/span><\/span><\/label><input type=\"checkbox\"  id=\"ez-toc-cssicon-toggle-item-69f3fbc8ad062\"  aria-label=\"Toggle\" \/><nav><ul class='ez-toc-list ez-toc-list-level-1 ' ><li class='ez-toc-page-1 ez-toc-heading-level-3'><a class=\"ez-toc-link ez-toc-heading-1\" href=\"https:\/\/www.delveinsight.com\/blog\/a-crispr-cas9-approach-for-gene-therapy-the-hot-new-rd-field\/#Only_a_hundred_and_fifty_years_have_passed_since_Gregor_Mendels_discovery_of_simple_Mendelian_inheritance_In_a_remarkably_short_amount_of_time_humans_have_achieved_such_impressive_feats_as_sequencing_the_entire_human_genome_and_gaining_understanding_of_the_causes_of_most_genetic_disease_Now_that_researchers_have_all_this_information_at_hand_the_focus_has_shifted_to_the_design_of_reagents_that_can_target_specific_genomic_sequences_The_rapid_advancement_of_genome-editing_techniques_holds_much_promise_for_the_field_of_human_gene_therapy_From_bacteria_to_model_organisms_and_human_cells_genome_editing_tools_such_as_zinc-finger_nucleases_ZNFs_TALENs_and_CRISPRCas9_have_been_successfully_used_to_manipulate_the_respective_genomes_with_unprecedented_precision_With_regard_to_human_gene_therapy_it_is_of_great_interest_to_test_the_feasibility_of_genome_engineering_because_of_their_ease_of_customization_and_high-efficient_site-specific_cleavages_that_could_potentially_be_used_to_treat_a_variety_of_human_genetic_disorders_such_as_hemoglobinopathies_primary_immunodeficiencies_and_cancer\" >Only a hundred and fifty years have passed since Gregor Mendel\u2019s discovery of simple Mendelian inheritance. In a remarkably short amount of time humans have achieved such impressive feats as sequencing the entire human genome and gaining understanding of the causes of most genetic disease. Now that researchers have all this information at hand, the focus has shifted to the design of reagents that can target specific genomic sequences. The rapid advancement of genome-editing techniques holds much promise for the field of human gene therapy. From bacteria to model organisms and human cells, genome editing tools such as zinc-finger nucleases (ZNFs), TALENs, and CRISPR\/Cas9 have been successfully used to manipulate the respective genomes with unprecedented precision. With regard to human gene therapy, it is of great interest to test the feasibility of genome engineering because of their ease of customization and high-efficient site-specific cleavages that could potentially be used to treat a variety of human genetic disorders such as hemoglobinopathies, primary immunodeficiencies, and cancer.<\/a><\/li><li class='ez-toc-page-1 ez-toc-heading-level-3'><a class=\"ez-toc-link ez-toc-heading-2\" href=\"https:\/\/www.delveinsight.com\/blog\/a-crispr-cas9-approach-for-gene-therapy-the-hot-new-rd-field\/#Unraveling_the_potential_of_CRISPR-Cas9_for_gene_therapy\" >Unraveling the potential of CRISPR-Cas9 for gene therapy<\/a><\/li><li class='ez-toc-page-1 ez-toc-heading-level-3'><a class=\"ez-toc-link ez-toc-heading-3\" href=\"https:\/\/www.delveinsight.com\/blog\/a-crispr-cas9-approach-for-gene-therapy-the-hot-new-rd-field\/#Patent_Litigation_over_control_of_the_revolutionary_CRISPR-Cas9_tech\" >Patent Litigation over control of the revolutionary CRISPR-Cas9 tech<\/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.delveinsight.com\/blog\/a-crispr-cas9-approach-for-gene-therapy-the-hot-new-rd-field\/#Gene_Therapys_fruition\" >Gene Therapy\u2019s fruition?<\/a><\/li><\/ul><\/nav><\/div>\n<h3 style=\"text-align:justify;\"><span class=\"ez-toc-section\" id=\"Only_a_hundred_and_fifty_years_have_passed_since_Gregor_Mendels_discovery_of_simple_Mendelian_inheritance_In_a_remarkably_short_amount_of_time_humans_have_achieved_such_impressive_feats_as_sequencing_the_entire_human_genome_and_gaining_understanding_of_the_causes_of_most_genetic_disease_Now_that_researchers_have_all_this_information_at_hand_the_focus_has_shifted_to_the_design_of_reagents_that_can_target_specific_genomic_sequences_The_rapid_advancement_of_genome-editing_techniques_holds_much_promise_for_the_field_of_human_gene_therapy_From_bacteria_to_model_organisms_and_human_cells_genome_editing_tools_such_as_zinc-finger_nucleases_ZNFs_TALENs_and_CRISPRCas9_have_been_successfully_used_to_manipulate_the_respective_genomes_with_unprecedented_precision_With_regard_to_human_gene_therapy_it_is_of_great_interest_to_test_the_feasibility_of_genome_engineering_because_of_their_ease_of_customization_and_high-efficient_site-specific_cleavages_that_could_potentially_be_used_to_treat_a_variety_of_human_genetic_disorders_such_as_hemoglobinopathies_primary_immunodeficiencies_and_cancer\"><\/span><span style=\"color:#a18405;\">Only a hundred and fifty years have passed since Gregor Mendel\u2019s discovery of simple Mendelian inheritance. In a remarkably short amount of time humans have achieved such impressive feats as sequencing the entire human genome and gaining understanding of the causes of most genetic disease. Now that researchers have all this information at hand, the focus has shifted to the design of reagents that can target specific genomic sequences. The rapid advancement of genome-editing techniques holds much promise for the field of human gene therapy. From bacteria to model organisms and human cells, genome editing tools such as zinc-finger nucleases (ZNFs), TALENs, and CRISPR\/Cas9 have been successfully used to manipulate the respective genomes with unprecedented precision. With regard to human gene therapy, it is of great interest to test the feasibility of genome engineering because of their ease of customization and high-efficient site-specific cleavages that could potentially be used to treat a variety of human genetic disorders such as hemoglobinopathies, primary immunodeficiencies, and cancer.<\/span><span class=\"ez-toc-section-end\"><\/span><\/h3>\n<h3><span class=\"ez-toc-section\" id=\"Unraveling_the_potential_of_CRISPR-Cas9_for_gene_therapy\"><\/span><span style=\"color:#2d2d69;\">Unraveling the potential of CRISPR-Cas9 for gene therapy<\/span><span class=\"ez-toc-section-end\"><\/span><\/h3>\n<p style=\"text-align:justify;\"><span style=\"color:#824965;\">The molecular machinery from the prokaryotic clustered regularly interspaced short palindromic repeats (CRISPR)-Cas immune system has broadly been repurposed for genome editing in eukaryotes. In particular, the sequence-specific Cas9 endonuclease can be flexibly harnessed for the genesis of precise double-stranded DNA breaks, using single guide RNAs that are readily programmable. The endogenous DNA repair machinery subsequently generates genome modifications, either by random insertion or deletions using non-homologous end joining (NHEJ), or designed integration of mutations or genetic material using homology-directed repair (HDR) templates. This technology has opened new avenues for the investigation of genetic diseases in general, and for gene therapy applications in particular.<\/span><\/p>\n<h3><span class=\"ez-toc-section\" id=\"Patent_Litigation_over_control_of_the_revolutionary_CRISPR-Cas9_tech\"><\/span><span style=\"color:#2d2d69;\">Patent Litigation over control of the revolutionary CRISPR-Cas9 tech<\/span><span class=\"ez-toc-section-end\"><\/span><\/h3>\n<p style=\"text-align:justify;\"><span style=\"color:#824965;\">Despite the predicted utility of a successful gene editing technique, many current methods like Zinc Fingers Nucleases and TALENs have confounding issues like low efficiency, time-consuming procedures, and lack of specificity for both model organisms and humans. In the past several years, a new gene editing system viz, CRISPR-Cas9 derived from bacteria, has arisen as a frontrunner for efficient and successful gene editing.<\/span><\/p>\n<p style=\"text-align:justify;\"><span style=\"color:#824965;\">Research in the area of CRISPR\/Cas9 is gaining speed and this system could very well be the solution to many medical issues we face today. For evidence of CRISPR\/Cas9&#8217;s promise, look no further than its attendant battle over intellectual property. Novartis and Atlas Venture joined together to form Editas Medicine, but a breakup of co-founders led Berkeley&#8217;s Jennifer Doudna to take her IP to the competing Intellia Therapeutics, while Swiss rival CRISPR Therapeutics has conflicting claims of its own backed by Versant. And now a team at Johns Hopkins has done some experiments to demonstrate its promise in engineering human stem cell therapies.<\/span><\/p>\n<p style=\"text-align:justify;\"><span style=\"color:#824965;\">This proves that gene editing has staggering potential and that it can be developed as a naturalistic method of correcting defective genes by getting at the underlying causes of a broad range of diseases.<\/span><\/p>\n<h3><span class=\"ez-toc-section\" id=\"Gene_Therapys_fruition\"><\/span><span style=\"color:#2d2d69;\"><strong>Gene Therapy\u2019s fruition?<\/strong><\/span><span class=\"ez-toc-section-end\"><\/span><\/h3>\n<p style=\"text-align:justify;\"><span style=\"color:#824965;\">The world of gene therapy in which single-dose treatments correct debilitating defects enjoyed something of a renaissance in 2014. Strong clinical results from leaders in the once-maligned field spurred renewed optimism, helping a new generation of startups secure millions in venture financing to develop their next-generation approaches to the field. And that led to something of a trickle-up phenomenon in the industry, as the innovations of biotechs and academics convinced the world&#8217;s biggest players to give this field a second look. Now Bayer, Pfizer, Biogen Idec and Astellas are among the many companies toiling in gene therapy, joining high-profile biotechs like bluebird bio and uniQure.<\/span><\/p>\n<p style=\"text-align:justify;\"><span style=\"color:#2d2d69;\"><strong>DelveInsight\u2019s Reports<\/strong> have already established a reputation of offering the affordable and comprehensive industry coverage and &#8220;on-the-ground&#8221; analysis in virtually every region of the world. These reports provide complete information for over 300 gene therapies which are in the pipeline for various therapy areas like; <span style=\"color:#ff0000;\"><em>Oncology, Genitourinary, Dermatology, Central nervous system, Genetic Disorders, Hematological disorders, Metabolic disorders, Ophthalmology, Cardiovascular disease, Respiratory , Immunology, <\/em><\/span>and many more&#8230;<\/span><\/p>\n<p style=\"text-align:justify;\"><span style=\"color:#2d2d69;\"><strong>DelveInsight\u2019s Gene Therapy Reports<\/strong> cover the entire gene therapy market scenario including technology assessments, licensing opportunities, collaborations, market trends, pipeline coverage and competitive landscape. The report essentially provides DelveInsight\u2019s proprietary market and pipeline analytics which identifies the front runners of all therapeutic areas. It also identifies the potential market movers and future regulatory landscape. These reports are designed to provide the clients with the means to out produce their competitors by developing a product that makes history.<\/span><\/p>\n<p><span style=\"color:#2d2d69;\">For more info on Gene Therapy Reports for various Therapeutic areas contact us at: <span style=\"text-decoration:underline;\"><strong><span style=\"color:#3366ff;\"><a style=\"color:#3366ff;text-decoration:underline;\" href=\"mailto:info@delveinsight.com\">info@delveinsight.com<\/a><\/span><\/strong><\/span>.<\/span><\/p>\n","protected":false},"excerpt":{"rendered":"<p>Only a hundred and fifty years have passed since Gregor Mendel\u2019s discovery of simple Mendelian inheritance. In a remarkably short amount of time humans have achieved such impressive feats as sequencing the entire human genome and gaining understanding of the causes of most genetic disease. Now that researchers have all this information at hand, the [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":0,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"_editorskit_title_hidden":false,"_editorskit_reading_time":3,"_editorskit_is_block_options_detached":false,"_editorskit_block_options_position":"{}","advgb_blocks_editor_width":"","advgb_blocks_columns_visual_guide":"","footnotes":""},"categories":[17],"tags":[173,180,209,258,267,356,386,453,479,558],"industry":[17225],"therapeutic_areas":[17235],"class_list":["post-352","post","type-post","status-publish","format-standard","hentry","category-articles","tag-collaborations","tag-competitive-landscape","tag-delveinsights-gene-therapy-reports","tag-gene-therapy","tag-genome-editing","tag-licensing-opportunities","tag-market-trends","tag-patent-litigation","tag-pipeline-coverage","tag-technology-assessments","industry-pharmaceutical","therapeutic_areas-cell-and-gene-therapy"],"acf":[],"yoast_head":"<!-- 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