[Cellular therapy of hereditary diseases].
This analytical review of the literature on cellular therapy of hereditary diseases summarizes results of their treatment by stem cell transplantation. It describes the main sources of stem cells and considers prospects of and limitations on the use of cell therapy for the management of hereditary disorders. Cell technologies are considered to be promising for the treatment of a variety of inherited diseases including metabolic disturbances, hemopathies, hematopoietic disorders, pulmonary diseases in children, bone and nervous diseases. Special attention is given to the use of genetically modified stem cells. An international register of patients is deemed necessary for the evaluation of outcomes of cellular therapy.
Bochkov
N P
NP
Nikitina
V A
VA
Roslova
T A
TA
Chaushev
I N
IN
Iakushina
I I
II
D000326:Adrenoleukodystrophy / Q000628:therapy; D000328:Adult; D016026:Bone Marrow Transplantation; D001997:Bronchopulmonary Dysplasia / Q000628:therapy; D002648:Child; D003550:Cystic Fibrosis / Q000628:therapy; D005198:Fanconi Syndrome / Q000628:therapy; D030342:Genetic Diseases, Inborn / Q000628:therapy*; D018380:Hematopoietic Stem Cell Transplantation; D006453:Hemoglobinopathies / Q000628:therapy; D006801:Humans; D007231:Infant, Newborn; D007965:Leukodystrophy, Globoid Cell / Q000628:therapy; D008059:Mucopolysaccharidosis I / Q000628:therapy; D039902:Multipotent Stem Cells / Q000637:transplantation; D009422:Nervous System Diseases / Q000628:therapy; D033581:Stem Cell Transplantation