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.

August 11, 2020
Open Access

Efficient in Utero Gene Transfer to the Mammalian Inner Ears by the Synthetic Adeno-Associated Viral Vector Anc80L65

Molecular Therapy. Methods & Clinical Development
Chin-Ju HuYen-Fu Cheng
August 11, 2020
Open Access

AAV-Genome Population Sequencing of Vectors Packaging CRISPR Components Reveals Design-Influenced Heterogeneity

Molecular Therapy. Methods & Clinical Development
Ngoc Tam TranPhillip W L Tai
August 12, 2020

miR-126a-3p targets HIF-1α and alleviates obstructive sleep apnea syndrome with hypertension

Human Cell
Lirong HeJiulong Kuang
September 10, 2020
Open Access

Gene-Editing Technologies Paired With Viral Vectors for Translational Research Into Neurodegenerative Diseases

Frontiers in Molecular Neuroscience
Joseph Edward RittinerBoris Kantor
September 12, 2020
Review

Direct convective delivery of adeno-associated virus gene therapy for treatment of neurological disorders

Journal of Neurosurgery
Russell R LonserKrystof S Bankiewicz
August 11, 2020
Open Access

Gene Therapy Preserves Retinal Structure and Function in a Mouse Model of NMNAT1 -Associated Retinal Degeneration

Molecular Therapy. Methods & Clinical Development
Scott H GreenwaldEric A Pierce
September 17, 2020

Rapid determination of full and empty adeno-associated virus capsid ratio by capillary isoelectric focusing

Current Molecular Medicine
Tingting LiAndras Guttman
September 11, 2020

Restoring the natural tropism of AAV2 vectors for human liver

Science Translational Medicine
Marti Cabanes-CreusLeszek Lisowski
August 20, 2020
Open Access

Gene editing and elimination of latent herpes simplex virus in vivo

Nature Communications
Martine AubertKeith R Jerome
August 14, 2020
Open Access

Injectable BMP-2 gene-activated scaffold for the repair of cranial bone defect in mice

Stem Cells Translational Medicine
Kai SunBing Wang
August 25, 2020
Open Access

Promoter Orientation within an AAV-CRISPR Vector Affects Cas9 Expression and Gene Editing Efficiency

The CRISPR Journal
Lewis E FryRobert E MacLaren
August 23, 2020
Review
Open Access

Adeno-Associated Virus Mediated Gene Therapy for Corneal Diseases

Pharmaceutics
Prabhakar BastolaMatthew L Hirsch
August 29, 2020
Open Access

AAV Gene Transfer to the Heart

Methods in Molecular Biology
Suya WangWilliam T Pu
August 11, 2020
Open Access

CRISPR-Cas9-Mediated In Vivo Gene Integration at the Albumin Locus Recovers Hemostasis in Neonatal and Adult Hemophilia B Mice

Molecular Therapy. Methods & Clinical Development
Qingnan WangYang Yang
August 29, 2020

Gene Therapy: Contest between Adeno-Associated Virus and Host Cells and the Impact of UFMylation

Molecular Pharmaceutics
Shubham Maurya, Giridhara R Jayandharan
August 28, 2020

Adeno-Associated Virus-Induced Dorsal Root Ganglion Pathology

Human Gene Therapy
Juliette HordeauxJames M Wilson

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