Gene Therapy Generates New Neurons to Treat Huntington's Disease
Researchers have developed a novel gene therapy to regenerate functional new neurons in mouse models of Huntington's disease
gene therapy
CRISPR-Edited Immune Cells Can Survive and Thrive after Infusion into Cancer Patients
New data on first-in-US trial sheds new light on success of gene editing and cell function
6 Patients with Rare Blood Disease Doing Well after Gene Therapy Clinical Trial
Treatment uses person's own stem cells instead of donor cells
Horizon Discovery to Provide Access to Novel Base Editing Technology
Next-generation gene editing system has applications in development of cell and gene therapies
Researchers Create Functional Mini-Liver by 3D Bioprinting
Technique developed at Human Genome and Stem Cell Research Center, funded by FAPESP and hosted by the University of São Paulo, produced hepatic tissue in the laboratory in only 90 days and could become an alternative to organ transplantation in future
Genomic Cut and Paste Using a Class 1 CRISPR System
Japanese researchers led by Osaka University show that a Class 1 CRISPR gene editing system can achieve functional DNA repairs in human cells with no prominent off-target effects
A New CRISPR-Cas9 Protein to Increase Precision of Gene Editing
New development could be useful for future gene therapies in humans, which require high precision
When Added to Gene Therapy, Plant-Based Compound May Enable Faster, More Effective Treatments
Scripps Research team finds that a nontoxic molecule closely related to resveratrol can overcome barriers to delivering gene therapy into stem cells
Can a DNA Construction Kit Replace Expensive Antibody Medication?
A study in animals similar in size to humans brings us a step closer to the clinical use of antibody gene therapy
BIA Separations Introduces Cornerstone AAV Process Development Service to Accelerate Gene Therapy Production
CORNERSTONE program integrates process development expertise and novel technology to remove development bottlenecks in the manufacture of Gene Therapy Medicinal Products (GTMPs)