Introduction to AAV as a Gene Therapy Vector, Part 2

Introduction

In the first part of our three part series introducing AAV as a gene therapy vector, we talked about basic AAV vector biology. In this post, we’re going to take a step back to answer the question of “Why AAV?” and look at some opportunities in the AAV engineering space. Viral vectors are one of the three main classes of gene therapy delivery vehicles. The other two are lipid nanoparticles (LNPs), now famous for their roles in mRNA vaccines, and plasmid electroporation. Relative to these other options, viral vectors have the advantage of higher delivery efficiency, better targeting, and a…

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related reading

• Introduction to AAV as a Gene Therapy Vector, Part 1substack.com
• Delivering gene therapy’s promise - Dyno Therapeuticsdynotx.substack.com
• Diversifying gene therapy vectors with machine learningsubstack.com
• Diversifying gene therapy vectors with machine learningsubstack.com
• Viral vector platforms within the gene therapy landscape | Signal Transduction and Targeted Therapynature.com
• CRISPR Gene Therapy: Applications, Limitations, and Implications for the Futurencbi.nlm.nih.gov
• Manufacture of Third-Generation Lentivirus for Preclinical Use, with Process Development Considerations for Translation to Good Manufacturing Practicencbi.nlm.nih.gov
• Clinical use of lentiviral vectors | Leukemianature.com
• Addgene: Lentiviral Vector Guideaddgene.org
• Siren Biotechnologysirenbiotechnology.com
• Dyno Therapeutics: The Capsids You Needcenturyofbio.com
• Significantly Enhancing Adult Intelligence With Gene Editing May Be Possible — LessWronglesswrong.com