期刊
BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE
卷 1866, 期 9, 页码 -出版社
ELSEVIER
DOI: 10.1016/j.bbadis.2020.165772
关键词
Neuronal ceroid lipofuscinoses; Batten disease; Gene therapy; Neurodegeneration; Pre-clinical studies; Clinical trials
资金
- EU [666918]
- UK Medical Research Council [MR/R015325/1, MR/S009434/1, MR/N026101/1, MR/S036784/1, MR/R025134/1]
- UK Action Medical Research [GN2485]
- Wellcome Trust [204841/Z/16/Z]
- MRC [MC_U12266B]
- Contratos de atraccion de talento de la Comunidad de Madrid modalidad 2 [2017-T2/BMD-5323]
- MRC [MR/R025134/1, MR/S009434/1, MR/R015325/1, MR/N026101/1] Funding Source: UKRI
The neuronal ceroid lipofuscinoses (NCLs), also known as Batten disease, are a group of rare monogenic neurodegenerative diseases predominantly affecting children. All NCLs are lethal and incurable and only one has an approved treatment available. To date, 13 NCL subtypes (CLN1-8, CLN10-14) have been identified, based on the particular disease-causing defective gene. The exact functions of NCL proteins and the pathological mechanisms underlying the diseases are still unclear. However, gene therapy has emerged as an attractive therapeutic strategy for this group of conditions. Here we provide a short review discussing updates on the current gene therapy studies for the NCLs.
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