Gene Therapy News and Research

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Gene therapy is an experimental technique that uses genes to treat or prevent disease. In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patient’s cells instead of using drugs or surgery.
S-pseudotyped VLPs offer promising COVID-19 vaccine platform

S-pseudotyped VLPs offer promising COVID-19 vaccine platform

Researchers compare developed carriers for delivery of genome editing tools with existing analogs

Researchers compare developed carriers for delivery of genome editing tools with existing analogs

UC Davis uses microneedles to deliver gene therapy for retinal diseases

UC Davis uses microneedles to deliver gene therapy for retinal diseases

RCas9 gene therapy clears toxic RNA buildup in mouse model of myotonic dystrophy

RCas9 gene therapy clears toxic RNA buildup in mouse model of myotonic dystrophy

Earlier treatment with steroids reduces adverse outcomes in patients with septic shock

Earlier treatment with steroids reduces adverse outcomes in patients with septic shock

Study may expedite the process to cure blindness and macular degeneration

Study may expedite the process to cure blindness and macular degeneration

Consortium compiles recommendations for measuring HIV reservoir size in cure-directed studies

Consortium compiles recommendations for measuring HIV reservoir size in cure-directed studies

CRISPR-based system suppresses genes related to AAV antibody production

CRISPR-based system suppresses genes related to AAV antibody production

AAV gene therapy often leads to dorsal root ganglion pathology, show studies

AAV gene therapy often leads to dorsal root ganglion pathology, show studies

New Gyrolab AAVX Titer Kit launched to support the development cell and gene therapies

New Gyrolab AAVX Titer Kit launched to support the development cell and gene therapies

Trinity College researchers to participate in gene-based clinical trial for motor neuron disease

Trinity College researchers to participate in gene-based clinical trial for motor neuron disease

New microgel drug delivery method could improve effectiveness of pancreatic islet transplants

New microgel drug delivery method could improve effectiveness of pancreatic islet transplants

A powerful tool for separation and sizing of virus mixtures

A powerful tool for separation and sizing of virus mixtures

Study offers hope for treatment of Krabbe disease

Study offers hope for treatment of Krabbe disease

Study examines safety and early outcomes of gene therapy for spinal muscular atrophy

Study examines safety and early outcomes of gene therapy for spinal muscular atrophy

Cobra Biologics successfully produced master cell banks for CombiGene’s gene therapy

Cobra Biologics successfully produced master cell banks for CombiGene’s gene therapy

Using engineered off-the-shelf therapeutic T cells to fight cancer

Using engineered off-the-shelf therapeutic T cells to fight cancer

New treatment approach strengthens motor function and muscle of aged mice

New treatment approach strengthens motor function and muscle of aged mice

Deciphering the largest CRISPR system

Deciphering the largest CRISPR system

Nanoparticle carriers expand the effectiveness of gene therapies for human eye diseases

Nanoparticle carriers expand the effectiveness of gene therapies for human eye diseases