换一批
换一批
第一作者：
Wei,Chen
第一单位：
Department of Biological Medicines, Fudan University School of Pharmacy, Shanghai 201203, China.
作者：
关键词
AADC, aromatic-l-amino-acidAAVs, adeno-associated virusesAD, Alzheimer's diseaseARSA, arylsulfatase AASOs, antisense oligonucleotidesASPA, aspartoacylaseAdeno-associated virusesAdv, adenovirusBBB, blood–brain barrierBCSFB, blood–cerebrospinal fluid barrierBRB, blood–retina barrierBip, glucose regulated protein 78CHOP, CCAAT/enhancer binding homologous proteinCLN6, ceroidlipofuscinosis neuronal protein 6CNS, central nervous systemCSF, cerebrospinal fluidCentral nervous systemDelivery routesER, endoplasmic reticulumFDA, U.S. Food and Drug AdministrationGAA, lysosomal acid α-glucosidaseGAD, glutamic acid decarboxylaseGDNF, glial derived neurotrophic factorGene therapyHD, Huntington's diseaseHSPGs, heparin sulfate proteoglycansHTT, mutant huntingtinIDS, iduronate 2-sulfataseLVs, retrovirus/lentivirusLamp2a, lysosomal-associated membrane protein 2aNGF, nerve growth factorNeurodegenerative disordersPD, Parkinson's diseasePGRN, ProgranulinPINK1, putative kinase 1PTEN, phosphatase and tensin homologRGCs, retinal ganglion cellsRNAi, RNA interferenceRPE, retinal pigmented epithelialSGSH, lysosomal heparan-N-sulfamidase geneSMN, survival motor neuronSOD, superoxide dismutaseSUMF, sulfatase-modifying factorTFEB, transcription factor EBTPP1, tripeptidyl peptidase 1TREM2, triggering receptor expressed on myeloid cells 2UPR, unfolded protein responseZFPs, zinc finger proteinsmTOR, mammalian target of rapamycinsiRNA, small interfering RNA
DOI
10.1016/j.apsb.2020.01.015
PMID
32963936
发布时间
2024-03-29
- 浏览6
Acta pharmaceutica Sinica. B
2020年10卷8期
1347-1359页
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