Title : CRISPR/Cas9 mediated gene correction ameliorates abnormal phenotypes in spinocerebellar ataxia type 3 patient-derived induced pluripotent stem cells.

Pub. Date : 2021 Sep 17

PMID : 34535635






2 Functional Relationships(s)
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Compound Name
Protein Name
Organism
1 The accumulation of the mutant ataxin-3 proteins carrying expanded polyglutamine (polyQ) leads to selective degeneration of neurons. polyglutamine ataxin 3 Homo sapiens
2 The accumulation of the mutant ataxin-3 proteins carrying expanded polyglutamine (polyQ) leads to selective degeneration of neurons. polyglutamine ataxin 3 Homo sapiens