Results 141 to 150 of about 396,665 (375)
Expressing Transgenes That Exceed the Packaging Capacity of Adeno-Associated Virus Capsids.
Human Gene Therapy Methods, 2016 Recombinant adeno-associated virus vectors (rAAV) are being explored as gene delivery vehicles for the treatment of various inherited and acquired disorders.
Kyle Chamberlain +2 more
semanticscholar +1 more source
Advanced Science, EarlyView.CRISPR/Cas9, while transformative, faces challenges in specificity, precision, delivery, accessibility, flexibility, and safety. This review addresses these limitations by highlighting strategies to reduce off‐target effects, exploring HDR‐based and alternative editing approaches, and evaluating advanced delivery mechanisms.
Muna Alariqi +8 more
wiley +1 more source
Genome Engineering Using Adeno-associated Virus: Basic and Clinical Research Applications.
Molecular Therapy, 2016 In addition to their broad potential for therapeutic gene delivery, adeno-associated virus (AAV) vectors possess the innate ability to stimulate homologous recombination in mammalian cells at high efficiencies.
T. Gaj, B. Epstein, D. Schaffer
semanticscholar +1 more source
Post‐Translational Modifications in Cilia and Ciliopathies
Advanced Science, EarlyView.This review synthesizes current understanding of post‐translational modifications (PTMs) in ciliary proteins and emphasizes their roles in ciliary formation, homeostasis, and signaling. This review also discusses the implication of PTM dysregulation in ciliopathies and explores therapeutic strategies targeting PTM‐modifying enzymes.
Jie Ran, Jun Zhou
wiley +1 more source
Deoxyribonucleic Acid of Adeno-associated Satellite Virus [PDF]
, 1969 Heather D. Mayor +2 more
openalex +1 more source
Adeno-Associated Virus Gene Therapy for Liver Disease
Human Gene Therapy, 2016 The field of adeno-associated virus (AAV) gene therapy has progressed rapidly over the past decade, with the advent of novel capsid serotype and organ-specific promoters, and an increasing understanding of the immune response to AAV administration.
Lisa M. Kattenhorn +6 more
semanticscholar +1 more source
From Code to Life: The AI‐Driven Revolution in Genome Editing
Advanced Science, EarlyView.This review explores AI‐driven advancements in genome editing, focusing on CRISPR optimization, enhanced targeting precision, reduced off‐target effects, and novel tool design. It addresses ethical challenges, data biases, and highlights future prospects in precision medicine, agriculture, and synthetic biology.
Zhidong Li +10 more
wiley +1 more source
Engineering a serum-resistant and thermostable vesicular stomatitis virus G glycoprotein for pseudotyping retroviral and lentiviral vectors. [PDF]
, 2013 Vesicular stomatitis virus G glycoprotein (VSV-G) is the most widely used envelope protein for retroviral and lentiviral vector pseudotyping; however, serum inactivation of VSV-G pseudotyped vectors is a significant challenge for in vivo gene delivery ...
Hwang, B-Y, Schaffer, DV
core
eLifeRecombinant adeno-associated viruses (rAAVs) are the predominant gene therapy vector. Several rAAV vectored therapies have achieved regulatory approval, but production of sufficient rAAV quantities remains difficult.
Nina K Jain +2 more
doaj +1 more source