Gene Therapy

The use of DNA as a technique for treating disease is a key feature of gene therapy. This type of treatment is being investigated to either replacement of a mutated gene with a healthy copy, silence a mutated gene that is dysfunctional or insert a novel gene to help fight disease. Discover the latest research on gene therapy here.

September 26, 2020

Exosome as a Natural Gene Delivery Vector for Cancer Treatment

Current Cancer Drug Targets
Prasad PofaliVaishali Londhe
September 17, 2020
Open Access

Gene editing for the treatment of Primary Immunodeficiency Diseases

Human Gene Therapy
Rajeev RaiAlessia Cavazza
September 19, 2020
Review
Open Access

Novel therapies for mucopolysaccharidosis type III

Journal of Inherited Metabolic Disease
Berna Seker YilmazJulien Baruteau
September 19, 2020

After Third Death, Audentes' AT132 Remains on Clinical Hold

Human Gene Therapy
Alex Philippidis
September 23, 2020

Production of Baculovirus and Stem Cells for Baculovirus-Mediated Gene Transfer into Human Mesenchymal Stem Cells

Methods in Molecular Biology
Friederike EiltsPeter Czermak
September 23, 2020
Review

CRISPR/Cas gene therapy

Journal of Cellular Physiology
Baohong Zhang
September 22, 2020
Review

Polymeric nano-carriers for on-demand delivery of genes via specific responses to stimuli

Journal of Materials Chemistry. B, Materials for Biology and Medicine
Khan MuhammadYakai Feng
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 23, 2020
Review
Open Access

Electroporation as the Immunotherapy Strategy for Cancer in Veterinary Medicine: State of the Art in Latin America

Vaccines
Felipe MagliettiGuillermo Marshall
September 23, 2020

Helper virus-free gutless adenovirus (HF-GLAd): a new platform for gene therapy

BMB Reports
Jida Liu, Dai-Wu Seol
September 18, 2020
Open Access

Rescuing AAV gene transfer from neutralizing antibodies with an IgG-degrading enzyme

JCI Insight
Zachary C ElmoreAravind Asokan
September 17, 2020

A drug-tunable Flt23k gene therapy for controlled intervention in retinal neovascularization

Angiogenesis
Jinying ChenGuei-Sheung Liu
September 25, 2020
Open Access

Replicate Aptima assay for quantifying residual plasma viremia in individuals on ART

Journal of Clinical Microbiology
Sonia BakkourReservoir Assay Validation and Evaluation Network (RAVEN)
September 26, 2020

Promoterless, nuclease-free genome editing confers a growth advantage for corrected hepatocytes in mice with methylmalonic acidemia

Hepatology : Official Journal of the American Association for the Study of Liver Diseases
Randy J ChandlerCharles P Venditti
September 22, 2020
Open Access

Rapid In-Process Monitoring of Lentiviral Vector Particles by High-Performance Liquid Chromatography

Molecular Therapy. Methods & Clinical Development
Julia TransfiguracionAmine A Kamen

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