AAV-based Gene Therapy

Adeno-associated virus (AAV)-based gene therapy is a biological vector that is being researched to be used as a potential therapeutic option. This gene therapy is designed to insert fragments of DNA into targeted cells to help treat diseases, such as hemophilia a. Discover the latest research on AAV-based gene therapy here.

February 23, 2021
Open Access

AAV9-mediated gene delivery of MCT1 to oligodendrocytes does not provide a therapeutic benefit in a mouse model of ALS.

Molecular Therapy. Methods & Clinical Development
Caroline EykensWim Robberecht
February 23, 2021
Open Access

Eliminating mesothelioma by AAV-vectored, PD1-based vaccination in the tumor microenvironment.

Molecular Therapy Oncolytics
Zhiwu TanZhiwei Chen
February 23, 2021

Novel AAV capsids for intravitreal gene therapy of photoreceptor disorders.

EMBO Molecular Medicine
Marina PavlouStylianos Michalakis
February 21, 2021

Long-term Stable Reduction of Low-density Lipoprotein in Nonhuman Primates Following In Vivo Genome Editing of PCSK9.

Molecular Therapy : the Journal of the American Society of Gene Therapy
Lili WangJames M Wilson
February 19, 2021
Open Access

Immunogenicity and inflammatory properties of respiratory syncytial virus attachment G protein in cotton rats.

PloS One
Margaret E MartinezStefan Niewiesk
February 20, 2021

rAAV-Mediated Human FGF-2 Gene Therapy Enhances Osteochondral Repair in a Clinically Relevant Large Animal Model Over Time In Vivo.

The American Journal of Sports Medicine
Yannik P MorscheidHenning Madry
February 18, 2021
Open Access

Evolving AAV-delivered therapeutics towards ultimate cures.

Journal of Molecular Medicine : Official Organ of the Gesellschaft Deutscher Naturforscher Und Ärzte
Xiangjun HeBo Feng
February 16, 2021

Ciliary neurotrophic factor overexpression protects the heart against pathological remodelling in angiotensin II-infused mice.

Biochemical and Biophysical Research Communications
Peng ZhongJianye Peng
February 14, 2021
Open Access

Development of an AAV9-RNAi-mediated silencing strategy to abrogate TRPM4 expression in the adult heart.

Pflügers Archiv : European journal of physiology
Rebekka MedertMarc Freichel
February 13, 2021

pNaSS-Grafted PCL Film-Guided rAAV TGF-β Gene Therapy Activates the Chondrogenic Activities in Human Bone Marrow Aspirates.

Human Gene Therapy
Jagadeesh Kumar VenkatesanMagali Cucchiarini
February 13, 2021

Viral Vector Delivery of DREADDs for CNS Therapy.

Current Gene Therapy
Ceri A Pickering, Nicholas D Mazarakis
February 13, 2021

Gene Therapy Strategies for Glaucomatous Neurodegeneration.

Current Gene Therapy
Rafael Lani-LouzadaHilda Petrs-Silva
February 13, 2021
Open Access

PSF functions as a repressor of hypoxia-induced angiogenesis by promoting mitochondrial function.

Cell Communication and Signaling : CCS
Lijie DongXiaorong Li
February 13, 2021
Open Access

Gene delivery to the hypoglossal motor system: preclinical studies and translational potential.

Gene Therapy
Brendan M DoyleDavid D Fuller
February 13, 2021

Deep diversification of an AAV capsid protein by machine learning.

Nature Biotechnology
Drew H BryantEric D Kelsic
February 13, 2021
Open Access

Visceral adipose tissue-directed FGF21 gene therapy improves metabolic and immune health in BTBR mice.

Molecular Therapy. Methods & Clinical Development
Nicholas J QueenLei Cao
February 13, 2021
Open Access

AAV-CRB2 protects against vision loss in an inducible CRB1 retinitis pigmentosa mouse model.

Molecular Therapy. Methods & Clinical Development
Thilo M BuckJan Wijnholds
February 13, 2021
Open Access

Nanopore sequencing of native adeno-associated virus (AAV) single-stranded DNA using a transposase-based rapid protocol.

NAR Genomics and Bioinformatics
Marco T RadukicJörn Kalinowski

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