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  2. Tay–Sachs disease - Wikipedia

    en.wikipedia.org/wiki/TaySachs_disease

    TaySachs disease is inherited in an autosomal recessive pattern. The HEXA gene is located on the long (q) arm of human chromosome 15, between positions 23 and 24. TaySachs disease is an autosomal recessive genetic disorder, meaning that when both parents are carriers, there is a 25% risk of giving birth to an affected child with each ...

  3. GM2 gangliosidoses - Wikipedia

    en.wikipedia.org/wiki/GM2_gangliosidoses

    The disease results from mutations on chromosome 5 in the HEXB gene, critical for the lysosomal enzymes beta-N-acetylhexosaminidase A and B. Sandhoff disease is clinically indistinguishable from TaySachs disease. The most common form, infantile Sandhoff disease, is usually fatal by early childhood. [5]

  4. Hexosaminidase - Wikipedia

    en.wikipedia.org/wiki/Hexosaminidase

    Over 100 different mutations have been discovered just in infantile cases of TaySachs disease alone. [11] The most common mutation, which occurs in over 80 percent of TaySachs patients, results from a four base pair addition (TATC) in exon 11 of the Hex A gene. This insertion leads to an early stop codon, which causes the Hex A deficiency ...

  5. GM2-gangliosidosis, AB variant - Wikipedia

    en.wikipedia.org/wiki/GM2-gangliosidosis,_AB_variant

    Signs and symptoms of GM2-gangliosidosis, AB variant are identical with those of infantile TaySachs disease, except that enzyme assay testing shows normal levels of hexosaminidase A. [2] Infantile Sandhoff disease has similar symptoms and prognosis, except that there is deficiency of both hexosaminidase A and hexosaminidase B. Infants with this disorder typically appear normal until the age ...

  6. HEXA - Wikipedia

    en.wikipedia.org/wiki/HEXA

    A “knockout” model, which is a mouse that has been genetically modified to observe the effects of inactivation of or damage to certain genes, found that the mice that were administered the HEXA gene experienced many of the same symptoms of Tay-Sachs, with one exception: GM2 buildup was distributed differently in the brains of the mice than ...

  7. Baby Has $5 Million Surgery to Remove Left Side of Brain at ...

    www.aol.com/baby-5-million-surgery-remove...

    Related: Mom Noticed Her Toddler Was Acting Strange.Days Later, She Found Out It Was Stage 4 Cancer (Exclusive) From there, things began to get worse. Andalusia recalls seeing Caper’s eye twitch ...

  8. Sandhoff disease - Wikipedia

    en.wikipedia.org/wiki/Sandhoff_disease

    Sandhoff disease is a lysosomal genetic, lipid storage disorder caused by the inherited deficiency to create functional beta-hexosaminidases A and B. [1] [2] These catabolic enzymes are needed to degrade the neuronal membrane components, ganglioside GM2, its derivative GA2, the glycolipid globoside in visceral tissues, [1] and some oligosaccharides.

  9. Substrate reduction therapy - Wikipedia

    en.wikipedia.org/wiki/Substrate_reduction_therapy

    TaySachs disease. The disease occurs when harmful quantities of a fatty acid derivative called a ganglioside accumulate in the nerve cells of the brain . Gangliosides are lipids , components of cellular membranes, and the ganglioside GM2, implicated in TaySachs disease, is especially common in the nervous tissue of the brain .