CRISPR/Cas9 technology in the modeling of and evaluation of possible treatments for Niemann-Pick C
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Publisher
Springer Science and Business Media LLC
Link
https://link.springer.com/content/pdf/10.1007/s11033-024-09801-1.pdf
Reference42 articles.
1. Vanier MT (2010) Niemann-Pick disease type C. Orphanet J Rare Dis 5(1):16
2. Vanier MT (2010) Niemann-Pick disease type C. Orphanet J Rare Dis 5:16
3. Greer WL, Riddell DC, Gillan TL, Girouard GS, Sparrow SM, Byers DM et al (1998) The Nova Scotia (type D) form of Niemann-Pick disease is caused by a G3097–>T transversion in NPC1. Am J Human Genet 63(1):52–54
4. Vance JE (2006) Lipid imbalance in the neurological disorder. Niemann-Pick C Dis FEBS Lett 580(23):5518–5524
5. Patterson MC, Mengel E, Wijburg FA, Muller A, Schwierin B, Drevon H et al (2013) Disease and patient characteristics in NP-C patients: findings from an international disease registry. Orphanet J Rare Dis 8(1):1–10
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