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AAV-based Gene Therapy

AAV-based Gene Therapy diagram by undefined

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
Advanced Materials

Thermosensitive Hydrogel Based on PEO-PPO-PEO Poloxamers for a Controlled In Situ Release of Recombinant Adeno-Associated Viral Vectors for Effective Gene Therapy of Cartilage Defects

Advanced MaterialsNovember 25, 2019
Henning MadryMagali Cucchiarini
1
Scientific Reports

Social defeat stress-specific increase in c-Fos expression in the extended amygdala in mice: Involvement of dopamine D1 receptor in the medial prefrontal cortex

Scientific ReportsNovember 13, 2019
Chisato NumaTomoyuki Furuyashiki
bioRxiv

Efficient Homology-directed Repair with Circular ssDNA Donors

bioRxivDecember 5, 2019
Sukanya IyerScot A. Wolfe
1
APL Bioengineering

AAV-mediated gene therapy targeting TRPV4 mechanotransduction for inhibition of pulmonary vascular leakage

APL BioengineeringDecember 1, 2019
Juan LiDonald E. Ingber
Circulation

In Vivo AAV-CRISPR/Cas9-Mediated Gene Editing Ameliorates Atherosclerosis in Familial Hypercholesterolemia

CirculationNovember 29, 2019
Huan ZhaoBin Zhou
2
Advanced Science

Gene Delivery to Nonhuman Primate Preimplantation Embryos Using Recombinant Adeno-Associated Virus

Advanced ScienceNovember 6, 2019
Dan WangWei-Zhi Ji
2
2
Gut

Growth differentiation factor 11 attenuates liver fibrosis via expansion of liver progenitor cells

GutNovember 25, 2019
Zhen DaiAmar Deep Sharma
8
Circulation Research

CRISPR-Mediated Activation of Endogenous Gene Expression in the Postnatal Heart

Circulation ResearchNovember 15, 2019
Eric SchogerLaura Cecilia Zelarayan
3
Journal of Neuromuscular Diseases

Advancements in AAV-mediated Gene Therapy for Pompe Disease

Journal of Neuromuscular DiseasesNovember 25, 2019
S M SalabarriaManuela Corti
Human Gene Therapy

AAV-mediated Gene Therapy for Aldehyde Dehydrogenase 2 Deficiency Reduces Esophageal DNA Damage and Adducts Associated with Chronic Ethanol Ingestion

Human Gene TherapyDecember 5, 2019
Yuki MatsumuraKatie M Stiles
Circulation. Heart Failure

Early Treatment of Coxsackievirus B3-Infected Animals With Soluble Coxsackievirus-Adenovirus Receptor Inhibits Development of Chronic Coxsackievirus B3 Cardiomyopathy

Circulation. Heart FailureNovember 1, 2019
Sandra PinkertHenry Fechner
Molecular Therapy : the Journal of the American Society of Gene Therapy

A Genetically Engineered Primary Human Natural Killer Cell Platform for Cancer Immunotherapy

Molecular Therapy : the Journal of the American Society of Gene TherapyOctober 15, 2019
Emily J PomeroyBranden S. Moriarity
18
9
International Journal of Molecular Sciences

rAAV Engineering for Capsid-Protein Enzyme Insertions and Mosaicism Reveals Resilience to Mutational, Structural and Thermal Perturbations

International Journal of Molecular SciencesNovember 14, 2019
Rebecca Christine FeinerKristian Mark Müller
Cell Reports

Anti-CRISPR AcrIIA5 Potently Inhibits All Cas9 Homologs Used for Genome Editing

Cell ReportsNovember 12, 2019
Bianca GarciaAlan R. Davidson
47
19
Methods in Molecular Biology

Continual Conscious Bioluminescent Imaging in Freely Moving Mice

Methods in Molecular BiologyNovember 14, 2019
Juan Antinao DiazRajvinder Karda
1
Wiley Interdisciplinary Reviews. Nanomedicine and Nanobiotechnology

Strategies for nonviral nanoparticle-based delivery of CRISPR/Cas9 therapeutics

Wiley Interdisciplinary Reviews. Nanomedicine and NanobiotechnologyDecember 2, 2019
Feng-Qian ChenQi Liu
The Journal of Thoracic and Cardiovascular Surgery

Effects of genetic transfection on calcium cycling pathways mediated by double-stranded adeno-associated virus in postinfarction remodeling

The Journal of Thoracic and Cardiovascular SurgerySeptember 30, 2019
Michael G KatzAnthony S Fargnoli
1
Journal of Clinical Medicine

AAV Mediated Delivery of Myxoma Virus M013 Gene Protects the Retina against Autoimmune Uveitis

Journal of Clinical MedicineNovember 29, 2019
Raela B RidleyCristhian J Ildefonso
Molecular Therapy : the Journal of the American Society of Gene Therapy

GPR108 Is a Highly Conserved AAV Entry Factor

Molecular Therapy : the Journal of the American Society of Gene TherapyNovember 13, 2019
Amanda M DudekLuk H. Vandenberghe
2
The Journal of Allergy and Clinical Immunology

Sensitization of spinal itch transmission neurons in a mouse model of chronic itch requires an astrocytic factor

The Journal of Allergy and Clinical ImmunologyNovember 21, 2019
Keisuke KogaMakoto Tsuda

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