Innovations in CRISPR-Based Therapies
Gene and cell therapies have shown tremendous advancement in the last 5 years. Prominent examples include the successful use of CRISPR-edited stem cells for treating blood disorders like sickle cell anemia and beta-thalassemia, and ongoing clinical trials for treating blindness. This mini-review ass...
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| Veröffentlicht in: | Molecular biotechnology 2023-02, Vol.65 (2), p.138-145 |
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| creator | Kesavan, Gokul |
| description | Gene and cell therapies have shown tremendous advancement in the last 5 years. Prominent examples include the successful use of CRISPR-edited stem cells for treating blood disorders like sickle cell anemia and beta-thalassemia, and ongoing clinical trials for treating blindness. This mini-review assesses the status of CRISPR-based therapies, both in vivo and ex vivo, and the challenges associated with clinical translation. In vivo CRISPR therapies have been used to treat eye and liver diseases due to the practicality of delivering editing components to the target tissue. In contrast, even though ex vivo CRISPR therapy involves cell isolation, expansion, and infusion, its advantages include characterizing the gene edits before infusion and restricting off-target effects in other tissues. Further, the safety, affordability, and feasibility of CRISPR therapies, especially for treating large number of patients, are discussed. |
| doi_str_mv | 10.1007/s12033-021-00411-x |
| format | Article |
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genetics</topic><topic>Anemia, Sickle Cell - therapy</topic><topic>Biochemistry</topic><topic>Biological Techniques</topic><topic>Biotechnology</topic><topic>Biotechnology industry</topic><topic>Blood diseases</topic><topic>Cell Biology</topic><topic>Cell Separation</topic><topic>Chemistry</topic><topic>Chemistry and Materials Science</topic><topic>Clinical trials</topic><topic>Consortia</topic><topic>CRISPR</topic><topic>CRISPR-Cas Systems</topic><topic>Gene Editing</topic><topic>Gene therapy</topic><topic>Genetic disorders</topic><topic>Genetic Therapy</topic><topic>Genomes</topic><topic>Hematological diseases</topic><topic>Human Genetics</topic><topic>Humans</topic><topic>Liver diseases</topic><topic>Muscular dystrophy</topic><topic>Mutation</topic><topic>Photoreceptors</topic><topic>Protein Science</topic><topic>Proteins</topic><topic>Review</topic><topic>Sickle cell disease</topic><topic>Stem cells</topic><topic>Thalassemia</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Kesavan, Gokul</creatorcontrib><collection>Medline</collection><collection>MEDLINE</collection><collection>MEDLINE (Ovid)</collection><collection>MEDLINE</collection><collection>MEDLINE</collection><collection>PubMed</collection><collection>CrossRef</collection><collection>ProQuest Central (Corporate)</collection><collection>Bacteriology Abstracts (Microbiology B)</collection><collection>Biotechnology Research Abstracts</collection><collection>Industrial and Applied Microbiology Abstracts (Microbiology A)</collection><collection>Virology and AIDS Abstracts</collection><collection>Health & Medical Collection</collection><collection>ProQuest Central (purchase pre-March 2016)</collection><collection>Biology Database (Alumni Edition)</collection><collection>Medical Database (Alumni Edition)</collection><collection>Science Database (Alumni Edition)</collection><collection>ProQuest Pharma Collection</collection><collection>Technology Research Database</collection><collection>ProQuest SciTech Collection</collection><collection>ProQuest Technology Collection</collection><collection>ProQuest Natural Science Collection</collection><collection>Hospital Premium Collection</collection><collection>Hospital Premium Collection (Alumni Edition)</collection><collection>ProQuest Central (Alumni) (purchase pre-March 2016)</collection><collection>Materials Science & Engineering Collection</collection><collection>ProQuest Central (Alumni Edition)</collection><collection>ProQuest Central UK/Ireland</collection><collection>ProQuest Central Essentials</collection><collection>Biological Science Collection</collection><collection>ProQuest Central</collection><collection>Technology Collection</collection><collection>Natural Science Collection</collection><collection>Environmental Sciences and Pollution Management</collection><collection>ProQuest One Community College</collection><collection>ProQuest Central Korea</collection><collection>Engineering Research Database</collection><collection>Health Research Premium Collection</collection><collection>Health Research Premium Collection (Alumni)</collection><collection>ProQuest Central Student</collection><collection>AIDS and Cancer Research Abstracts</collection><collection>SciTech Premium Collection</collection><collection>ProQuest Health & Medical Complete (Alumni)</collection><collection>ProQuest Engineering Collection</collection><collection>ProQuest Biological Science Collection</collection><collection>Health & Medical Collection (Alumni Edition)</collection><collection>Medical Database</collection><collection>Science Database</collection><collection>Algology Mycology and Protozoology Abstracts (Microbiology C)</collection><collection>Biological Science Database</collection><collection>Engineering Database</collection><collection>Biotechnology and BioEngineering Abstracts</collection><collection>ProQuest One Academic Eastern Edition (DO NOT USE)</collection><collection>ProQuest One Academic</collection><collection>ProQuest One Academic UKI Edition</collection><collection>Engineering Collection</collection><collection>ProQuest Central Basic</collection><jtitle>Molecular biotechnology</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Kesavan, Gokul</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Innovations in CRISPR-Based Therapies</atitle><jtitle>Molecular biotechnology</jtitle><stitle>Mol Biotechnol</stitle><addtitle>Mol Biotechnol</addtitle><date>2023-02-01</date><risdate>2023</risdate><volume>65</volume><issue>2</issue><spage>138</spage><epage>145</epage><pages>138-145</pages><issn>1073-6085</issn><eissn>1559-0305</eissn><abstract>Gene and cell therapies have shown tremendous advancement in the last 5 years. 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| ispartof | Molecular biotechnology, 2023-02, Vol.65 (2), p.138-145 |
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| subjects | Anemia Anemia, Sickle Cell - genetics Anemia, Sickle Cell - therapy Biochemistry Biological Techniques Biotechnology Biotechnology industry Blood diseases Cell Biology Cell Separation Chemistry Chemistry and Materials Science Clinical trials Consortia CRISPR CRISPR-Cas Systems Gene Editing Gene therapy Genetic disorders Genetic Therapy Genomes Hematological diseases Human Genetics Humans Liver diseases Muscular dystrophy Mutation Photoreceptors Protein Science Proteins Review Sickle cell disease Stem cells Thalassemia |
| title | Innovations in CRISPR-Based Therapies |
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