Tag Archives: Gene

Blind Man’s Sight Partially Restored with ‘Optogenetics’ Gene Therapy

Advertisement Continue reading the main story Supported by Continue reading the main story Matter Scientists Partially Restored a Blind Man’s Sight With New Gene Therapy Using a technique called optogenetics, researchers added light-sensitive proteins to the man’s retina, giving him a blurry view of objects. In an experiment, a 58-year-old volunteer with a genetic form… Read More »

Orbit Subretinal Delivery System for Gene Therapy: Interview with Mike Keane, CTO of Gyroscope Therapeutics

Gyroscope Therapeutics, a retinal gene therapy company, has developed the Orbit subretinal delivery system (SDS). The technology is designed to deliver therapies to the retina without the need for invasive procedures, such as vitrectomies, which involve removing the gel-like substance within the eye, or retinotomies, which involve making a hole in the retina. The company… Read More »

NIH researchers discover gene for rare disease of excess bone tissue growth

What Researchers at the National Institutes of Health have discovered a second gene that causes melorheostosis, a rare group of conditions involving an often painful and disfiguring overgrowth of bone tissue. The gene, SMAD3, is part of a pathway that regulates cell development and growth. The researchers are now working to develop an animal model with… Read More »

Modifier Gene May Explain Why Some with Cystic Fibrosis are Less Prone to Infection

Cystic fibrosis is caused by an inherited mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Due to this mutation, the CFTR protein doesn’t embed in cell membranes to form a channel for chlorine ions the way it should. As a result, mucus-producing cells secrete a thicker-than-normal mucus that can create blockages in the lungs and… Read More »

Schizophrenia risk gene linked to cognitive deficits in mice

Researchers have discovered in mice how one of the few genes definitively linked to schizophrenia, called SETD1A, likely confers risk for the illness. Mice genetically engineered to lack a functioning version of the enzyme-coding gene showed abnormalities in working memory, mimicking those commonly seen in schizophrenia patients. Restoring the gene’s function corrected the working memory… Read More »

Many Genes Influence Same-Sex Sexuality, Not a Single ‘Gay Gene’

How do genes influence our sexuality? The question has long been fraught with controversy. An ambitious new study — the largest ever to analyze the genetics of same-sex sexual behavior — found that genetics does play a role, responsible for perhaps a third of the influence on whether someone has same-sex sex. The influence comes… Read More »

Commentary: Novartis CEO: Gene therapy offers hope of cures in a treatment, but US needs new pricing, payment model

We live in an incredible era of progress in human health. Advances in cell and gene therapies are beginning to yield powerful new treatments for some of the most devastating illnesses. With many of these new therapies we are no longer combating diseases over time, but potentially curing them in a single treatment. This is… Read More »