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Second-generation gene therapy can increase protein synthesis and secretion
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Minimizing cellular stress improves durability of liver-targeted gene therapy
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Peer review, open access, comprehensive groundbreaking review
ASC Therapeutics is a privately held biopharmaceutical company developing transformative in vivo gene replacement, gene editing and allogeneic cell therapies for hematological, metabolic and other rare diseases. Peer-reviewed open access publication “Gene Therapy for Hemophilia A: Current and Next Generation Approaches” Biotherapy Expert Opinion1. This publication provides an update on the scientific and clinical progress of gene replacement therapy for hemophilia A, focusing on:
This press release features multimedia. Check out the full version here: https://www.businesswire.com/news/home/20220110005741/en/
Professor Steven W. Pipe, University of Michigan. (Photo: Business Wire)
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Current and next-generation approaches to achieve safe, durable and stable transgene expression while avoiding the challenges of factor VIII replacement therapy;
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A comprehensive review of current and past clinical studies of gene therapy in patients with hemophilia A;
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Addresses the critical role of viral constructs in reducing therapeutic doses and minimizing cellular stress, inducing unfolded protein responses, and causing loss of protein production in hepatocytes;
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Second-generation gene therapy incorporating chimeric DNA sequences into transgenes can increase the synthesis and secretion of coagulation factors and improve the efficacy, safety, and durability of gene replacement therapy for hemophilia A and other coagulation disorders.
“This…
The full story can be found on Benzinga.com
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