Strategies for Smuggling Gene Therapies Past the Immune System
Strategies for Smuggling Gene Therapies Past the Immune System
Researchers are devising ways to prevent viral vectors carrying gene therapies from triggering an immune response.
Strategies for Smuggling Gene Therapies Past the Immune System
Strategies for Smuggling Gene Therapies Past the Immune System

Researchers are devising ways to prevent viral vectors carrying gene therapies from triggering an immune response.

Researchers are devising ways to prevent viral vectors carrying gene therapies from triggering an immune response.

gene therapy
Stem Cell Funding Agency CIRM Is Nearly Out of Funds
Stem Cell Funding Agency CIRM Is Nearly Out of Funds
Chia-Yi Hou | Jul 8, 2019
The California Institute for Regenerative Medicine has been funding research since it formed in 2004, giving out nearly $3 billion in grants to date.
FDA Approves Gene Therapy for Spinal Muscular Atrophy
FDA Approves Gene Therapy for Spinal Muscular Atrophy
Ashley Yeager | May 27, 2019
At $2 million for a single dose, Novartis’s Zolgensma is the most expensive medicine to date, but still less expensive over a lifetime than another approved drug for the rare genetic disease.
Image of the Day: Cone Preservation
Image of the Day: Cone Preservation
Chia-Yi Hou | Apr 30, 2019
Researchers treat mice with retinitis pigmentosa using a gene therapy that reduces cone loss.
Gene Therapy Effective for Severe Combined Immunodeficiency
Gene Therapy Effective for Severe Combined Immunodeficiency
Shawna Williams | Apr 17, 2019
Researchers report they’ve found a way to restore immune function in infants with one form of “bubble boy disease.”
Preliminary Results Point to Success of In Vivo Gene Editing
Preliminary Results Point to Success of In Vivo Gene Editing
Carolyn Wilke | Feb 12, 2019
Two studies show signs that the introduced DNA is functioning, but it’s too early to know if patients actually benefit.
CRISPR Editing Heads Off Disease in Mouse LiversCRISPR Editing Heads Off Disease in Mouse Livers
CRISPR Editing Heads Off Disease in Mouse Livers
Shawna Williams | Oct 9, 2018
Separate proof-of-concept studies report success against two inherited diseases.
Early Results of First In Vivo Gene-Editing Trial Encouraging
Early Results of First In Vivo Gene-Editing Trial Encouraging
Kerry Grens | Sep 5, 2018
Two patients who received Sangamo’s zinc finger–based treatment for Hunter syndrome have lower biomarkers of the condition, but no signs of new enzyme production.
Putting Exosomes to Work
Putting Exosomes to Work
Ruth Williams | Sep 1, 2018
Researchers identify a handy tool for tinkering with the versatile vesicles.
Infographic: Directing Exosome Traffic
Infographic: Directing Exosome Traffic
Ruth Williams | Sep 1, 2018
Researchers harness a protein to send cargo-carrying exosomes to mouse muscles.