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.
Mice live longer with nasal gene therapy

Mice live longer with nasal gene therapy

Study highlights the economic burden affecting individuals with sickle cell disease

Study highlights the economic burden affecting individuals with sickle cell disease

Postnatal gene therapy may be able to prevent or reverse deadly effects of Pitt-Hopkins syndrome

Postnatal gene therapy may be able to prevent or reverse deadly effects of Pitt-Hopkins syndrome

Gene therapy effectively reduces neuropathic pain in mice with spinal cord injuries

Gene therapy effectively reduces neuropathic pain in mice with spinal cord injuries

Study uses brain organoids to reveal how autism-linked genetic mutation disrupts neural development

Study uses brain organoids to reveal how autism-linked genetic mutation disrupts neural development

Better understanding of hydrocephalus could lead to improved treatments

Better understanding of hydrocephalus could lead to improved treatments

Exploring strategies for successful delivery of messenger RNA therapeutics

Exploring strategies for successful delivery of messenger RNA therapeutics

Bioprinting for bone repair enhanced with two growth factor encoding genes

Bioprinting for bone repair enhanced with two growth factor encoding genes

Experimental tool can precisely pick out interactions between immune cell and target antigen

Experimental tool can precisely pick out interactions between immune cell and target antigen

VectorBuilder to expand with $500 million ‘Gene Delivery Research and Manufacturing Campus

VectorBuilder to expand with $500 million ‘Gene Delivery Research and Manufacturing Campus

Let’s Talk about Parkinson’s

Let’s Talk about Parkinson’s

MOFs could help improve the delivery of genetic cargo into target cells

MOFs could help improve the delivery of genetic cargo into target cells

$4 million program launched to find transformative therapies for ADNP syndrome

$4 million program launched to find transformative therapies for ADNP syndrome

UI neuroscientists identify fundamental biochemical mechanism underlying memory storage

UI neuroscientists identify fundamental biochemical mechanism underlying memory storage

A single gene can control brain cell growth in humans, research shows

A single gene can control brain cell growth in humans, research shows

Sneaking large drug-carrying biological molecules into cells using a novel drug delivery system

Sneaking large drug-carrying biological molecules into cells using a novel drug delivery system

Synthetic viral vector and surgical approach enable efficient gene transfer into the inner ear of primates

Synthetic viral vector and surgical approach enable efficient gene transfer into the inner ear of primates

Bioactive small molecules enhance CRISPR/Cas9 loss-of-function editing in human cells

Bioactive small molecules enhance CRISPR/Cas9 loss-of-function editing in human cells

Researchers secure $12 million grant to develop improved therapies for hemophilia

Researchers secure $12 million grant to develop improved therapies for hemophilia

Combating COVID cytokine storm with targeted nucleic acid therapies

Combating COVID cytokine storm with targeted nucleic acid therapies