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Tay-Sachs, Canavan, GM1 and Sandhoff diseases

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Latest Tay Sachs Disease News and Research

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.
$28 million grant supports Cincinnati Children's role as coordinating center for Rare Diseases Clinical Research Network
The federal agency that oversees a large network of research centers seeking cures for hundreds of rare, disabling and often fatal diseases has named Cincinnati Children's as its new data management and coordinating center.

Latest Sandhoff Disease News and Research

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.
Researchers describe important step toward gene therapy for patients with Sandhoff disease
Babies with the rare, deadly genetic disorder Sandhoff disease begin to miss developmental milestones just months after birth. Lacking muscle tone, they never learn to sit up, develop heads too large to lift and eventually suffer uncontrollable seizures. There is no cure.

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.

Worse Than COVID? The Tasmanian Devil’s Contagious Cancer

It’s hard to imagine anything worse than the horrors at our hospitals right now. But in a recent JAMA webinar, Nicholas Christakis…

The post Worse Than COVID? The Tasmanian Devil’s Contagious Cancer appeared first on DNA Science.
Thinking About Thanksgiving? A Reminder of What COVID-19 Can Do To A Human Body

My blog posts around Thanksgiving are predictably dull: Turkey Genetics 101, The Peaceable Genomes of Pumpkins. But 2020 is like no other…

The post Thinking About Thanksgiving? A Reminder of What COVID-19 Can Do To A Human Body appeared first on DNA Science.
Genetic Clues in the Goop of a COVID Swab

The components of certain things are meant to remain mysterious. The ingredients of sausage. A burger’s slimy secret sauce. The recipe for…

The post Genetic Clues in the Goop of a COVID Swab 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.