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Tay-Sachs, Canavan, GM1 and Sandhoff diseases

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Executive Leadership Transition
After 14 years, Sue Kahn plans to step down. Read a letter from Sue and learn more about the position. Read More
Passage Bio's First Patient Dosed
The first patient has been dosed in Imagine-1 study of PBGM01 gene therapy for infantile GM1 gangliosidosis. Read More
Lysogene Clinical Trial
FDA Fast Track Designation for LYS-GM101 Gene Therapy for the Treatment of GM1. Read More

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News

Latest Tay Sachs Disease News and Research

A new combination therapy for treating rare, fatal Sanfilippo syndrome in children
A study aiming to develop a new therapeutic technique could bring a revolution in our approach to treating rare, fatal Sanfilippo syndrome, a disorder that affects children as young as 2 years old and leads to childhood dementia and premature death.
Study underscores the need for more precise rare disease definitions
Some diseases are like black swans. They occur so rarely that many physicians never encounter them in their clinical practice, complicating efforts to treat them.
New CRISPR genome “prime editing” system
Gene editing to correct genetic faults and removing the risk of hereditary diseases may soon become reality. Researchers have come up with a new gene editing technology that could help patients with sickle cell anaemia and up 89 percent of other genetic defects.

Latest Sandhoff Disease News and Research

A new combination therapy for treating rare, fatal Sanfilippo syndrome in children
A study aiming to develop a new therapeutic technique could bring a revolution in our approach to treating rare, fatal Sanfilippo syndrome, a disorder that affects children as young as 2 years old and leads to childhood dementia and premature death.
New drug lures stem cells to damaged tissue, improves treatment efficacy
Scientists at Sanford Burnham Prebys Medical Discovery Institute have created a drug that can lure stem cells to damaged tissue and improve treatment efficacy--a scientific first and major advance for the field of regenerative medicine.
Gene therapy is the best stratagem to impact the course of GM2 gangliosidosis
Since its clinical description in 1881, Tay-Sachs disease has had almost totemic significance as a cruel and relentless genetic condition that destroys the developing brain of babies and young children.

Latest Canavan Disease (Leukodystrophies) News and Research

Human stem cells show promise against fatal children's diseases
Scientists have used human stem cells to dramatically improve the condition of mice with a neurological condition similar to a set of diseases in children that are invariably fatal, according to an article in the June issue of the journal Cell Stem Cell.

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Geneticist Ricki Lewis blogs from the cutting edge of genomics, including genetic testing, stem cells, gene therapy and more.

Katla on Netflix: Volcanoes, Glaciers, and Meteorites, Oh My!

I usually write about a sci fi book or film midsummer. That’s more necessary this summer than ever, when science reality &ndash…

The post Katla on Netflix: Volcanoes, Glaciers, and Meteorites, Oh My! appeared first on DNA Science.
Looking Into a COVID Lung Using Spatial Transcriptomics

As the early weeks of the pandemic unfolded and health care workers struggled to save so many lives, researchers began tracking the…

The post Looking Into a COVID Lung Using Spatial Transcriptomics appeared first on DNA Science.
Hulu’s False Positive Misses the Mark in Evoking Rosemary’s Baby

I looked forward to Hulu’s original horror film False Positive, pitched as a modern-day Rosemary’s Baby. It premiered at the Tribeca Film…

The post Hulu’s False Positive Misses the Mark in Evoking Rosemary’s Baby appeared first on DNA Science.

Latest Gangliosidosis News and Research

Gene therapy is the best stratagem to impact the course of GM2 gangliosidosis
Since its clinical description in 1881, Tay-Sachs disease has had almost totemic significance as a cruel and relentless genetic condition that destroys the developing brain of babies and young children.
New gene therapy shows promising results for treating neurodegenerative disorders
A new gene therapy approach designed to replace the enzyme that is deficient in patients with the inherited neurodegenerative disorders Tay-Sachs and Sandhoff diseases successfully delivered the therapeutic gene to the brains of treated mice, restored enzyme function, and extended survival by about 2.5-fold.
Researchers devise new genetic technique for IVF embryos
Researchers at the Johns Hopkins University School of Medicine have devised a new technique, which helps couples that are affected by or are carriers of genetic diseases have in vitro fertilized babies free of both the disease in question and other chromosomal abnormalities. The results were reported in the April issue of Fertility and Sterility.