He also discovered the pattern of the possibility that Tay-Sachs could be passed down through family links, more commonly (at the time) of those in the eastern Jewish population.
There is a pilot trial currently open to pregnant women at risk for having children with fatal metabolic disorders including Tay-Sachs and Sandhoff diseases. The trial is being conducted out of Duke University Medical Center in Durham, N.C.
tay-sachs research paper | Neurology | Clinical Medicine
- Tay-Sachs Disease Treatment research papers discuss that there is no known treatment yet, but there is progress being made in finding a way to treat this disease.
Explain Tay-Sachs disease. – Order Research Paper
Children born with Tay-Sachs disease lack a vital enzyme, this substance accumulates abnormally and causes progressive damage until the nervous system can no longer sustain life. Researchers in the Consortium have refined a viral vector carrying two human genes- Hex-A, which is deficient in Tay-Sachs disease, and Hex-B, which is deficient in Sandhoff disease. They have successfully injected these genes into the brains of laboratory mice, prompting production of the missing enzyme at levels sufficient to correct the deficiency. However, curing affected mice is just one very promising step in their research.
Tay Sachs Disease Essay Examples | Kibin
The next task for the Consortium is to translate the results into advances in a large animal model of the disease, specifically cats. A cat’s brain is seen as an intermediary between mouse and human in terms of size and complexity. Al of this will hopefully translate into the ultimate goal of the Consortium- to develop a clinical trial of the gene therapy safe enough for humans within the next four years. If successful, it could potentially stop the progression of Tay-Sachs disease, although it would not be able to undo damage already done to the affected individuals.
Tay Sachs Disease Essay Examples
Eventually the damaged caused by the accumulation of toxic levels of GM2 gangliosides leads to the destruction of neurons, which causes the signs and symptoms of Tay- Sachs disease and at last causing death of the patient.
A Paper on What Is Tay-Sachs Disease
Without Hex- A, there is a buildup of gangliosides in the cells of the brain, causing major damage to the cells (“Learning About Tay-Sachs Disease”)....