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Global gene and cell replacement strategies via stem cells

Authors
 K.I Park  ;  J Ourednik  ;  V Ourednik  ;  RM Taylor  ;  KS Aboody  ;  KI Auguste  ;  MB Lachyankar  ;  DE Redmond  ;  EY Snyder 
Citation
 GENE THERAPY, Vol.9(10) : 613-624, 2002 
Journal Title
GENE THERAPY
ISSN
 0969-7128 
Issue Date
2002
MeSH
Adult ; Amyloidosis/therapy ; Animals ; Brain Ischemia/therapy ; Brain Neoplasms/therapy ; Central Nervous System/cytology* ; Genetic Therapy/methods* ; Hematopoietic Stem Cell Transplantation/methods* ; Humans ; Intellectual Disability/therapy ; Models, Neurological* ; Nerve Degeneration/therapy ; Nerve Regeneration* ; Trauma, Nervous System/therapy*
Keywords
neural transplantation ; neuroregeneration and repair ; development ; mental retardation ; neurodegeneration ; gene therapy
Abstract
The inherent biology of neural stem cells (NSCs) endows them with capabilities that not only circumvent many of the limitations of other gene transfer vehicles, but that enable a variety of novel therapeutic strategies heretofore regarded as beyond the purview of neural transplantation. Most neurodegenerative diseases are characterized not by discrete, focal abnormalities but rather by extensive, multifocal, or even global neuropathology. Such widely disseminated lesions have not conventionally been regarded as amenable to neural transplantation. However, the ability of NSCs to engraft diffusely and become integral members of structures throughout the host CNS, while also expressing therapeutic molecules, may permit these cells to address that challenge. Intriguingly, while NSCs can be readily engineered to express specified foreign genes, other intrinsic factors appear to emanate spontaneously from NSCs and, in the context of reciprocal donor-host signaling, seem to be capable of neuroprotective and/or neuroregenerative functions. Stem cells additionally have the appealing ability to 'home in' on pathology, even over great distances. Such observations help to advance the idea that NSCs - as a prototype for stem cells from other solid organs - might aid in reconstructing the molecular and cellular milieu of maldeveloped or damaged organs.
Files in This Item:
T200204774.pdf Download
DOI
10.1038/sj/gt/3301721
Appears in Collections:
1. College of Medicine (의과대학) > Dept. of Pediatrics (소아과학교실) > 1. Journal Papers
Yonsei Authors
Park, Kook In(박국인) ORCID logo https://orcid.org/0000-0001-8499-9293
URI
https://ir.ymlib.yonsei.ac.kr/handle/22282913/143755
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