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.

June 17, 2020

Neutralisation of adeno-associated virus transduction by human vitreous humour

Gene Therapy
Sławomir AndrzejewskiChristopher J Layton
June 4, 2020

Attenuation of Heparan Sulfate Proteoglycan Binding Enhances In Vivo Transduction of Human Primary Hepatocytes with AAV2

Molecular Therapy. Methods & Clinical Development
Marti Cabanes-CreusLeszek Lisowski
May 23, 2020
Review

Precision Therapy for Inherited Retinal Disease: At the Forefront of Genomic Medicine

Clinics in Laboratory Medicine
Nicole Koulisis, Aaron Nagiel
June 4, 2020

PDK1 regulates the maintenance of cell body and the development of dendrites of purkinje cells by pS6 and PKCγ

The Journal of Neuroscience : the Official Journal of the Society for Neuroscience
Rui LiuJun Gao
June 15, 2020

Voretigene Neparvovec: A Review in RPE65 Mutation-Associated Inherited Retinal Dystrophy

Molecular Diagnosis & Therapy
Connie Kang, Lesley J Scott
June 17, 2020
Review
Open Access

WFH State-of-the-art paper 2020: In vivo lentiviral vector gene therapy for haemophilia

Haemophilia : the Official Journal of the World Federation of Hemophilia
Alessio Cantore, Luigi Naldini
June 13, 2020

Modeling and Rescue of RP2 Retinitis Pigmentosa Using iPSC-Derived Retinal Organoids

Stem Cell Reports
Amelia LaneMichael E Cheetham
June 12, 2020

Rescue of Advanced Pompe Disease in Mice with Hepatic Expression of Secretable Acid α-Glucosidase

Molecular Therapy : the Journal of the American Society of Gene Therapy
Umut CaginFederico Mingozzi
June 3, 2020
Open Access

Uncertainty in an era of transformative therapy for haemophilia: Addressing the unknowns

Haemophilia : the Official Journal of the World Federation of Hemophilia
Glenn F Pierce
May 30, 2020

AAV9-Mediated Expression of SMN Restricted to Neurons Does Not Rescue the Spinal Muscular Atrophy Phenotype in Mice

Molecular Therapy : the Journal of the American Society of Gene Therapy
Aurore BesseMaria Grazia Biferi
June 24, 2020
Review

Moving from the Bench Towards a Large Scale, Industrial Platform Process for Adeno-Associated Viral Vector Purification

Biotechnology and Bioengineering
Benjamin AdamsAndrew D Tustian
June 2, 2020

Protection against SIV in Rhesus Macaques Using Albumin and CD4-Based Vector-Mediated Gene Transfer

Molecular Therapy. Methods & Clinical Development
Sergei SpitsinSteven D Douglas

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