Feasibility of gene therapy for late neuronal ceroid lipofuscinosis. Review uri icon

Overview

MeSH

  • Adult
  • Aminopeptidases
  • Animals
  • Child
  • Child, Preschool
  • Clinical Trials as Topic
  • Dipeptidyl-Peptidases and Tripeptidyl-Peptidases
  • Genetic Vectors
  • Humans
  • Serine Proteases
  • Stem Cell Transplantation

MeSH Major

  • Endopeptidases
  • Genetic Therapy
  • Neuronal Ceroid-Lipofuscinoses

abstract

  • Late infantile neuronal ceroid lipofuscinosis is a progressive childhood neurodegenerative disorder characterized by intracellular accumulation of autofluorescent material resembling lipofuscin in neuronal cells. This report summarizes the new therapies under consideration for late infantile neuronal ceroid lipofuscinosis, with a focus on strategies for in vivo gene therapy for the retinal and central nervous system manifestations of the disease.

publication date

  • November 2001

has subject area

  • Adult
  • Aminopeptidases
  • Animals
  • Child
  • Child, Preschool
  • Clinical Trials as Topic
  • Dipeptidyl-Peptidases and Tripeptidyl-Peptidases
  • Endopeptidases
  • Genetic Therapy
  • Genetic Vectors
  • Humans
  • Neuronal Ceroid-Lipofuscinoses
  • Serine Proteases
  • Stem Cell Transplantation

Research

keywords

  • Journal Article
  • Review

Identity

Language

  • eng

PubMed ID

  • 11708986

Additional Document Info

start page

  • 1793

end page

  • 1798

volume

  • 58

number

  • 11