Clinical pharmacology perspective on development of adeno‐associated virus vector‐based retina gene therapy

JL Ford, E Karatza, H Mody… - Clinical …, 2024 - Wiley Online Library
Adeno‐associated virus (AAV) vector‐based gene therapy is an innovative modality being
increasingly investigated to treat diseases by modifying or replacing defective genes or …

A quantitative systems pharmacology (QSP) platform for preclinical to clinical translation of in-vivo CRISPR-Cas therapy

DA Desai, S Schmidt, R Cristofoletti - Frontiers in Pharmacology, 2024 - frontiersin.org
Background: In-vivo CRISPR Cas genome editing is a complex therapy involving lipid
nanoparticle (LNP), messenger RNA (mRNA), and single guide RNA (sgRNA). This novel …

[HTML][HTML] Gene Therapy in the Light of Lifestyle Diseases: Budesonide, Acetaminophen and Simvastatin Modulates rAAV Transduction Efficiency

Ż Słyk, N Stachowiak, M Małecki - Pharmaceuticals, 2024 - mdpi.com
Recombinant AAV (rAAV) vectors are increasingly favored for gene therapy due to their
useful features of vectorology, such as transfection of dividing and nondividing cells, the …