Cellular therapy for childhood neurodegenerative disease. Part I: Rationale and preclinical studies

Daniel J. Guillaume, Stephen L. Huhn, Nathan Selden, Robert D. Steiner

Research output: Contribution to journalArticle

19 Citations (Scopus)

Abstract

Successful cellular replacement in the diseased human central nervous system (CNS) faces numerous hurdles. In this first installment of a 2-part review, the authors report on the preclinical challenges involved in preparing for a major Phase I trial investigating the safety of human neural stem cell transplantation in a lysosomal storage disorder. Specifically, they discuss choice of the ideal disease for treatment, best donor cell type and source for implantation, the in vitro and in vivo methods used to estimate safety and efficacy, the challenges to noninvasive tracking of cells after transplantation, and the unique issues related to the immunology of CNS cellular transplantation.

Original languageEnglish (US)
Article numberE21
JournalNeurosurgical Focus
Volume24
Issue number3-4
DOIs
StatePublished - Mar 2008

Fingerprint

Neurodegenerative Diseases
Safety
Neural Stem Cells
Central Nervous System Diseases
Cell Transplantation
Stem Cell Transplantation
Allergy and Immunology
Central Nervous System
Transplantation
Therapeutics
In Vitro Techniques

Keywords

  • Cell replacement therapy
  • Central nervous system cell transplantation
  • Human stem cell
  • Neural stem cel
  • Neurodegenerative diease
  • Restorative neurosurgery

ASJC Scopus subject areas

  • Surgery
  • Clinical Neurology

Cite this

Cellular therapy for childhood neurodegenerative disease. Part I : Rationale and preclinical studies. / Guillaume, Daniel J.; Huhn, Stephen L.; Selden, Nathan; Steiner, Robert D.

In: Neurosurgical Focus, Vol. 24, No. 3-4, E21, 03.2008.

Research output: Contribution to journalArticle

Guillaume, Daniel J. ; Huhn, Stephen L. ; Selden, Nathan ; Steiner, Robert D. / Cellular therapy for childhood neurodegenerative disease. Part I : Rationale and preclinical studies. In: Neurosurgical Focus. 2008 ; Vol. 24, No. 3-4.
@article{9dc751a4d7e64adfb4f725d9d1e7f0d8,
title = "Cellular therapy for childhood neurodegenerative disease. Part I: Rationale and preclinical studies",
abstract = "Successful cellular replacement in the diseased human central nervous system (CNS) faces numerous hurdles. In this first installment of a 2-part review, the authors report on the preclinical challenges involved in preparing for a major Phase I trial investigating the safety of human neural stem cell transplantation in a lysosomal storage disorder. Specifically, they discuss choice of the ideal disease for treatment, best donor cell type and source for implantation, the in vitro and in vivo methods used to estimate safety and efficacy, the challenges to noninvasive tracking of cells after transplantation, and the unique issues related to the immunology of CNS cellular transplantation.",
keywords = "Cell replacement therapy, Central nervous system cell transplantation, Human stem cell, Neural stem cel, Neurodegenerative diease, Restorative neurosurgery",
author = "Guillaume, {Daniel J.} and Huhn, {Stephen L.} and Nathan Selden and Steiner, {Robert D.}",
year = "2008",
month = "3",
doi = "10.3171/FOC/2008/24/3-4/E20",
language = "English (US)",
volume = "24",
journal = "Neurosurgical Focus",
issn = "1092-0684",
publisher = "American Association of Neurological Surgeons",
number = "3-4",

}

TY - JOUR

T1 - Cellular therapy for childhood neurodegenerative disease. Part I

T2 - Rationale and preclinical studies

AU - Guillaume, Daniel J.

AU - Huhn, Stephen L.

AU - Selden, Nathan

AU - Steiner, Robert D.

PY - 2008/3

Y1 - 2008/3

N2 - Successful cellular replacement in the diseased human central nervous system (CNS) faces numerous hurdles. In this first installment of a 2-part review, the authors report on the preclinical challenges involved in preparing for a major Phase I trial investigating the safety of human neural stem cell transplantation in a lysosomal storage disorder. Specifically, they discuss choice of the ideal disease for treatment, best donor cell type and source for implantation, the in vitro and in vivo methods used to estimate safety and efficacy, the challenges to noninvasive tracking of cells after transplantation, and the unique issues related to the immunology of CNS cellular transplantation.

AB - Successful cellular replacement in the diseased human central nervous system (CNS) faces numerous hurdles. In this first installment of a 2-part review, the authors report on the preclinical challenges involved in preparing for a major Phase I trial investigating the safety of human neural stem cell transplantation in a lysosomal storage disorder. Specifically, they discuss choice of the ideal disease for treatment, best donor cell type and source for implantation, the in vitro and in vivo methods used to estimate safety and efficacy, the challenges to noninvasive tracking of cells after transplantation, and the unique issues related to the immunology of CNS cellular transplantation.

KW - Cell replacement therapy

KW - Central nervous system cell transplantation

KW - Human stem cell

KW - Neural stem cel

KW - Neurodegenerative diease

KW - Restorative neurosurgery

UR - http://www.scopus.com/inward/record.url?scp=42149170223&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=42149170223&partnerID=8YFLogxK

U2 - 10.3171/FOC/2008/24/3-4/E20

DO - 10.3171/FOC/2008/24/3-4/E20

M3 - Article

C2 - 18341399

AN - SCOPUS:42149170223

VL - 24

JO - Neurosurgical Focus

JF - Neurosurgical Focus

SN - 1092-0684

IS - 3-4

M1 - E21

ER -