Splicing-based Gene Editing for Efficient AAV Gene Therapies
Expediting gene therapies at the research level can require several essential tools, such as molecular cloning, plasmids, and viral vector systems, all of which must be carefully coordinated in order to design and produce functional therapies. To minimize the number of therapeutic products to be developed, one option is to deliver all the components required for gene editing by a single vector with an excellent safety and efficacy profile.
AAV (adeno-associated virus) meets these requirements, except that its effective packaging capacity is only ~3,600 nucleotides, which is incompatible with most editing strategies. Therefore, designing efficient vectors is critical for gene therapy research.
This webcast will highlight editing by spliceosome-mediated RNA trans-splicing, which circumvents the AAV packaging limit problem. RNA trans-splicing molecules require only ~200 nucleotides for activity, leaving over 3,000 nucleotides that can be expressed by the edited mRNA. Another strategy discussed will be designing efficient vectors that optimize the viral capsid and enable efficient cell entry while minimizing immune responses. These splicing-based strategies can be used to ensure safety and potency in both in vitro and in vivo settings.
About the Presenters
Dr. Llyod Mitchell
CEO and CSO, Splice Therapeutics
Dr, James Cody
Associate Director, Technical Sales and Evaluations,
Charles River Laboratories
nAAVigation® Accelerates Gene Therapy Programs
Established AAV platform has the capability to cut a viral vector gene therapy program timeline to GMP by more than half versus traditional process development.
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