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.
Study demonstrates the concept of systemic gene therapy treatment for gamma-sarcoglycanopathy

Study demonstrates the concept of systemic gene therapy treatment for gamma-sarcoglycanopathy

Newly approved gene therapy for SMA is based on delivery vehicle discovered by Penn expert

Newly approved gene therapy for SMA is based on delivery vehicle discovered by Penn expert

Children's Tumor Foundation establishes significant research initiative to combat NF2

Children's Tumor Foundation establishes significant research initiative to combat NF2

Newly discovered molecules safely halt the gene editing process

Newly discovered molecules safely halt the gene editing process

WVU researcher studies stubborn blood disease that strikes children

WVU researcher studies stubborn blood disease that strikes children

Researchers develop stem cell-based model to study neuron resilience and vulnerability in ALS

Researchers develop stem cell-based model to study neuron resilience and vulnerability in ALS

CU Anschutz researchers identify new immunodeficiency disease

CU Anschutz researchers identify new immunodeficiency disease

Discovery of small-molecule inhibitors could enable precise control over CRISPR-Cas9 genome editing

Discovery of small-molecule inhibitors could enable precise control over CRISPR-Cas9 genome editing

Sartorius Stedim Biotech introduces new ambr 15 cell culture microbioreactor system

Sartorius Stedim Biotech introduces new ambr 15 cell culture microbioreactor system

Potential stem cell therapies moving from lab bench to clinical application

Potential stem cell therapies moving from lab bench to clinical application

Will gene-editing of human embryos ever be justifiable?

Will gene-editing of human embryos ever be justifiable?

Gene repair improves seizure and memory in adult mouse models of autism

Gene repair improves seizure and memory in adult mouse models of autism

UT Southwestern researchers determine how circulating 'bad cholesterol' enters artery walls

UT Southwestern researchers determine how circulating 'bad cholesterol' enters artery walls

Yposkesi chairman to speak on ‘Manufacturing and the CDMO Perspective’ at Cell and Gene Meeting

Yposkesi chairman to speak on ‘Manufacturing and the CDMO Perspective’ at Cell and Gene Meeting

Reviving the heart's regenerative capacities using microRNAs

Reviving the heart's regenerative capacities using microRNAs

Study compares effectiveness and cost of gene therapy and HSCT in major beta-thalassemia

Study compares effectiveness and cost of gene therapy and HSCT in major beta-thalassemia

New gene therapy cures babies with fatal 'Bubble Boy' disease

New gene therapy cures babies with fatal 'Bubble Boy' disease

Researchers identify cause of inherited metabolic disorder

Researchers identify cause of inherited metabolic disorder

Key gene could decrease detrimental brain swelling after stroke

Key gene could decrease detrimental brain swelling after stroke

INTERPHEX 2019 Celebrates 40 years of Delivering Technology Innovation to Industry

INTERPHEX 2019 Celebrates 40 years of Delivering Technology Innovation to Industry