PMID- 11332775
OWN - NLM
STAT- MEDLINE
DCOM- 20010816
LR  - 20211203
IS  - 0065-2660 (Print)
IS  - 0065-2660 (Linking)
VI  - 45
DP  - 2001
TI  - Outlook for future treatment.
PG  - 217-24
AB  - Currently, no treatment is available for neuronal ceroid lipofuscinoses. The 
      progress of human genome project will stimulate molecular cloning of unidentified 
      genes underlying the NCLs, which will lead eventually clinical management and 
      therapies for NCL. Characterizing the native substrate(s) for the 
      palmitoyl-protein thioesterase-1 (PPT1) and tripeptidyl peptidase 1 (TPP1), 
      understanding the protein functions encoded by CLN genes, and uncovering the 
      pathological metabolic mechanism for the NCLs are the bases of designing rational 
      treatments for the NCLs. Testing potential therapeutic agents, replacing 
      deficient enzymes, and developing gene therapy will be the major tasks for NCL 
      researchers.
FAU - Zhong, N
AU  - Zhong N
AD  - Department of Human Genetics, New York State Institute for Basic Research in 
      Developmental Disabilities, Staten Island 10314, USA. omrddzhong@AOL.com
FAU - Wisniewski, K E
AU  - Wisniewski KE
LA  - eng
PT  - Journal Article
PT  - Review
PL  - United States
TA  - Adv Genet
JT  - Advances in genetics
JID - 0370421
RN  - 0 (Membrane Proteins)
RN  - 0 (Tripeptidyl-Peptidase 1)
RN  - EC 3.4.14.9 (TPP1 protein, human)
SB  - IM
MH  - Clinical Trials as Topic
MH  - Cloning, Molecular
MH  - Genetic Therapy
MH  - Humans
MH  - Lysosomes/enzymology
MH  - Membrane Proteins/genetics/metabolism
MH  - Neuronal Ceroid-Lipofuscinoses/genetics/metabolism/*therapy
MH  - Tripeptidyl-Peptidase 1
RF  - 27
EDAT- 2001/05/03 10:00
MHDA- 2001/08/17 10:01
CRDT- 2001/05/03 10:00
PHST- 2001/05/03 10:00 [pubmed]
PHST- 2001/08/17 10:01 [medline]
PHST- 2001/05/03 10:00 [entrez]
AID - 10.1016/s0065-2660(01)45013-9 [doi]
PST - ppublish
SO  - Adv Genet. 2001;45:217-24. doi: 10.1016/s0065-2660(01)45013-9.