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AAV-based Gene Therapy

AAV-based Gene Therapy diagram by Public Domain, Wikimedia
Public Domain, Wikimedia

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.

Top 20 most recent papers
The New England Journal of Medicine

Multiyear Follow-up of AAV5-hFVIII-SQ Gene Therapy for Hemophilia A

The New England Journal of MedicineJanuary 2, 2020
K John PasiWing Y Wong
Circulation

Cytokine mRNA Degradation in Cardiomyocytes Restrains Sterile Inflammation in Pressure Overloaded Hearts

CirculationJanuary 15, 2020
Shigemiki OmiyaKinya Otsu
1
bioRxiv

AAV Ablates Neurogenesis in the Adult Murine Hippocampus

bioRxivJanuary 19, 2020
Stephen T JohnstonMatthew Shtrahman
172
bioRxiv

SMALL ALPHAHERPESVIRUS LATENCY-ASSOCIATED PROMOTERS DRIVE EFFICIENT AND LONG-TERM TRANSGENE EXPRESSION IN THE CENTRAL NERVOUS SYSTEM

bioRxivDecember 31, 2019
Carola J MaturanaEsteban A Engel
7
Molecular Therapy : the Journal of the American Society of Gene Therapy

Gene Therapy for Glaucoma by Ciliary Body Aquaporin 1 Disruption Using CRISPR-Cas9

Molecular Therapy : the Journal of the American Society of Gene TherapyJanuary 26, 2020
Jiahui WuColin J Chu
3
Scientific Reports

Capsid-specific removal of circulating antibodies to adeno-associated virus vectors

Scientific ReportsJanuary 23, 2020
Berangere BertinFederico Mingozzi
1
Science Translational Medicine

Gene therapy delivering a paraoxonase 1 variant offers long-term prophylactic protection against nerve agents in mice

Science Translational MedicineJanuary 24, 2020
Venkaiah BetapudiNageswararao Chilukuri
1
1
Molecular Therapy : the Journal of the American Society of Gene Therapy

Single SERCA2a Therapy Ameliorated Dilated Cardiomyopathy for 18 Months in a Mouse Model of Duchenne Muscular Dystrophy

Molecular Therapy : the Journal of the American Society of Gene TherapyJanuary 26, 2020
Nalinda B WasalaDongsheng Duan
2
Molecular Therapy : the Journal of the American Society of Gene Therapy

Immune Responses to Viral Gene Therapy Vectors

Molecular Therapy : the Journal of the American Society of Gene TherapyJanuary 23, 2020
Jamie L ShirleyRoland W Herzog
1
Oncology Reports

Combination of AAV‑mediated NUPR1 knockdown and trifluoperazine induces premature senescence in human lung adenocarcinoma A549 cells in nude mice

Oncology ReportsJanuary 11, 2020
Yanzhe LiZhenyi Ma
1
1
Molecular Therapy : the Journal of the American Society of Gene Therapy

Muscle-Directed Delivery of an AAV1 Vector Leads to Capsid-Specific T Cell Exhaustion in Nonhuman Primates and Humans

Molecular Therapy : the Journal of the American Society of Gene TherapyJanuary 27, 2020
Gwladys GernouxChristian Mueller
Nature Communications

Single AAV-mediated mutation replacement genome editing in limited number of photoreceptors restores vision in mice

Nature CommunicationsJanuary 26, 2020
Koji M NishiguchiToru Nakazawa
2
The AAPS Journal

Recommendations for the Development of Cell-Based Anti-Viral Vector Neutralizing Antibody Assays

The AAPS JournalJanuary 8, 2020
Gorovits BorisShobha Purushothama
Molecular Therapy. Methods & Clinical Development

Inclusion of PF68 Surfactant Improves Stability of rAAV Titer when Passed through a Surgical Device Used in Retinal Gene Therapy

Molecular Therapy. Methods & Clinical DevelopmentJanuary 1, 2020
Maria I PatrícioRobert E MacLaren
4
Zhong nan da xue xue bao. Yi xue ban = Journal of Central South University. Medical sciences

Alzheimer's disease and nerve growth factor gene therapy

Zhong nan da xue xue bao. Yi xue ban = Journal of Central South University. Medical sciencesJanuary 24, 2020
Zhonghua HuangJie Zhang
Human Gene Therapy

Adeno associated virus serotype

Human Gene TherapyJanuary 10, 2020
Lauriel Freya EarleyR Jude Samulski
Nature Biomedical Engineering

Cytosine and adenine base editing of the brain, liver, retina, heart and skeletal muscle of mice via adeno-associated viruses

Nature Biomedical EngineeringJanuary 16, 2020
Jonathan M LevyDavid R Liu
174
1
Molecular Therapy. Nucleic Acids

Endogenous MicroRNA Competition as a Mechanism of shRNA-Induced Cardiotoxicity

Molecular Therapy. Nucleic AcidsJanuary 14, 2020
Meredith M CoursePaul N Valdmanis
Molecular Therapy. Methods & Clinical Development

Pathogenesis of Hepatic Tumors following Gene Therapy in Murine and Canine Models of Glycogen Storage Disease

Molecular Therapy. Methods & Clinical DevelopmentJanuary 1, 2020
Hye-Ri KangDwight D Koeberl
2
Molecular Therapy. Methods & Clinical Development

7T MRI Predicts Amelioration of Neurodegeneration in the Brain after AAV Gene Therapy

Molecular Therapy. Methods & Clinical DevelopmentJanuary 24, 2020
Heather L Gray-EdwardsDouglas R Martin
2

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