Altasciences

Issue 37 — Quantitative PCR and Droplet Digital: Analysis for Your Gene Therapy Programs


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In this issue, we will review the applications of PCR analysis for your gene therapy programs, and present two case studies.
Click to read this audiobook: https://www.altasciences.com/sites/default/files/2023-10/the-altascientist-issue-37-pcr_4.pdf
Gene therapy continues to accelerate through preclinical and clinical research arenas. These programs are developed with targeted and personalized medicines in mind. The goal of gene therapy is to safely deliver and incorporate a genetic alteration to restore or repair the protein of a missing or faulty gene. Preclinical assessments of gene therapies consider the general absorption, distribution, metabolism, and excretion of the drug, as well as data from tailored assessments to evaluate delivery and cell incorporation.
Gene therapies require, by design and definition, DNA and/or RNA delivery and analysis. While most ongoing research involves gene therapies being delivered in vivo via adeno-associated viral (AAV) vectors, new in vivo delivery mechanisms are on the rise, such as other types of delivery vectors including lipid nanoparticles.
CHAPTESR:
- 0:05 — About Issue 37
- 1:20 — Introduction to qPCR, dPCR, and RT-PCR
- 5:58 — Utility of qPCR, ddPCR, and RT-PCR
- 8:19 — PCR Applications
- 10:01 — Advantages and Applications
- 17:45 — Case Study 1
- 20:20 — Case Study 2
- 22:47 — Conclusion
About Altasciences:
Altasciences is an integrated drug development solution company offering pharmaceutical and biotechnology companies a proven, flexible approach to preclinical and clinical pharmacology studies, including formulation, manufacturing, and analytical services. For over 25 years, Altasciences has been partnering with sponsors to help support educated, faster, and more complete early drug development decisions. Altasciences’ integrated, full-service solutions include preclinical safety testing, clinical pharmacology and proof of concept, bioanalysis, program management, medical writing, biostatistics, clinical monitoring, and data management, all customizable to specific sponsor requirements. Altasciences helps sponsors get better drugs to the people who need them, faster.
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