Identification of New Modulators and Inhibitors of Palmitoyl-Protein Thioesterase 1 for CLN1 Batten Disease and Cancer-Reference-Cited by-同舟云学术

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Identification of New Modulators and Inhibitors of Palmitoyl-Protein Thioesterase 1 for CLN1 Batten Disease and Cancer

Published:2024-02-28 Issue:10 Volume:9 Page:11870-11882
ISSN:2470-1343
Container-title:ACS Omega
language:en
Short-container-title:ACS Omega

Author:

Puhl Ana C.¹,Raman Renuka¹,Havener Tammy M.²^ORCID,Minerali Eni¹,Hickey Anthony J.²³,Ekins Sean¹^ORCID

Affiliation:

1. Collaborations Pharmaceuticals, Inc., 840 Main Campus Drive, Lab 3510, Raleigh, North Carolina 27606, United States

2. UNC Catalyst for Rare Diseases, Eshelman School of Pharmacy, University of North Carolina at Chapel Hill, Chapel Hill, North Carolina 27599, United States

3. RTI International, Research Triangle Park, North Carolina 27709, United States

Funder

National Institute of Neurological Disorders and Stroke

National Center for Complementary and Integrative Health

National Institute of General Medical Sciences

Publisher

American Chemical Society (ACS)

Link

https://pubs.acs.org/doi/pdf/10.1021/acsomega.3c09607

Reference79 articles.

1. Lysosomal disorders: From storage to cellular damage

2. Lysosomal Biogenesis in Lysosomal Storage Disorders

3. Emptying the stores: lysosomal diseases and therapeutic strategies

4. Mice deficient in the lysosomal enzyme palmitoyl-protein thioesterase 1 (PPT1) display a complex retinal phenotype

5. The novel Cln1R151X mouse model of infantile neuronal ceroid lipofuscinosis (INCL) for testing nonsense suppression therapy

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