Cord blood and bone marrow transplantation in inherited metabolic diseases: scientific basis, current status and future directions

British Journal of Haematology
Vinod K Prasad, Joanne Kurtzberg

Abstract

Progressive degeneration of the central nervous system leading to the loss of neuromotor, neurophysiological and cognitive abilities is the fundamental clinical problem in patients with many inherited metabolic diseases (IMD). Worldwide experience shows that morbidity, quality of life, and survival in these patients can be improved by allogeneic haematopoietic stem cell transplantation (HSCT), particularly when performed early in the course of the disease. At present, while available for some conditions, exogenous enzyme replacement therapy is unable to correct cognitive and central nervous system disease because of its inability to cross the blood-brain barrier. In contrast, HSCT allows donor-derived, enzyme-producing cells to migrate to the brain and other organs providing a permanent enzyme replacement therapy. HSCT may also mediate non-hematopoietic cell regeneration or repair. Traditionally, bone marrow has been the graft source for IMD patients. However, in the last 5 years many studies utilizing unrelated donor umbilical cord blood (UCB) as a graft source have demonstrated that UCB provides rapid and increased access to transplantation with favourable outcomes. This review describes preclinical studies and past and prese...Continue Reading

References

Jan 1, 1992·Journal of Inherited Metabolic Disease·A VellodiK Hugh-Jones
Feb 6, 1986·The New England Journal of Medicine·P WongS Yoshida
Nov 28, 1987·British Medical Journal·A VellodiK Hugh-Jones
Oct 1, 1987·Archives of Disease in Childhood·A WillR F Stevens
Feb 6, 1986·The New England Journal of Medicine·J G WatsonA D Pearson
Jul 3, 1986·The New England Journal of Medicine·W E Fowler, S Boyarsky
Sep 1, 1969·Proceedings of the National Academy of Sciences of the United States of America·J C FratantoniE F Neufeld
Jul 10, 1970·Science·E F Neufeld, J C Fratantoni
Aug 1, 1971·Proceedings of the National Academy of Sciences of the United States of America·A G KnudsonJ E Curtis
Feb 1, 1971·Proceedings of the National Academy of Sciences of the United States of America·N Di FerranteR K Berglund
Jan 1, 1995·Journal of Inherited Metabolic Disease·N GuffonP Guibaud
Jan 1, 1995·Journal of Inherited Metabolic Disease·G V CoppaP Di Natale
Apr 1, 1995·The American Journal of Medicine·J Somani, R A Larson
Apr 12, 1994·Proceedings of the National Academy of Sciences of the United States of America·S U WalkleyS Wurzelmann
Apr 15, 1993·American Journal of Medical Genetics·C B WhitleyJ H Kersey
Jul 1, 1996·The Journal of Pediatrics·E J McKinnisC R Scott
Apr 17, 1998·The New England Journal of Medicine·W KrivitL A Lockman
Jul 22, 1998·The Journal of Pediatrics·N GuffonP Guibaud
Aug 26, 1998·The Journal of Pediatrics·D A WallS Kotagal
Mar 10, 1999·Journal of Inherited Metabolic Disease·E HerskhovitzA Vellodi
Jul 10, 1999·Journal of Inherited Metabolic Disease·A VellodiJ E Wraith
Sep 1, 2000·Bone Marrow Transplantation·A M YeagerH W Moser
Oct 24, 2001·Neurology·T LönnqvistU M Saarinen-Pihkala
Jan 5, 2002·Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation·H S GoodwinD A Wall
Aug 24, 2002·Science·Raymond F CastroH David Shine
Mar 7, 2003·Bone Marrow Transplantation·C PetersUNKNOWN Working Party on Inborn Errors, European Bone Marrow Transplant Group

❮ Previous
Next ❯

Citations

Jul 12, 2011·Journal of Inherited Metabolic Disease·Elizabeth A BraunlinRoberto Giugliani
Apr 12, 2011·International Journal of Hematology·Hiroshi YagasakiHideo Mugishima
Jan 27, 2012·Pediatric Research·Jos DomenJignesh Dalal
Feb 9, 2013·Pediatric Critical Care Medicine : a Journal of the Society of Critical Care Medicine and the World Federation of Pediatric Intensive and Critical Care Societies·Christine N DuncanUNKNOWN Pediatric Acute Lung Injury and Sepsis (PALISI) Network
Oct 24, 2012·Bone Marrow Research·Aleksandra Klimczak, Andrzej Lange
Aug 1, 2010·Therapeutic Advances in Endocrinology and Metabolism·Gregory M Pastores
May 27, 2014·Brain Research·Donald G Phinney, Iryna A Isakova
Jul 22, 2014·IUBMB Life·Laura Segatori
Nov 2, 2013·Neurologic Clinics·Gregory M Pastores, Gustavo H B Maegawa
Jul 18, 2012·Cytotherapy·David L Digiusto, Hans-Peter Kiem
May 8, 2010·Clinical Immunology : the Official Journal of the Clinical Immunology Society·Roberto Francese, Paolo Fiorina
Sep 19, 2015·Critical Reviews in Oncology/hematology·Stijn Vanhee, Bart Vandekerckhove
Oct 11, 2011·Transfusion·Belinda PopeRoss Grant
Jan 13, 2011·British Journal of Haematology·Melissa RhodesHaydar Frangoul
Aug 15, 2014·Pediatric Hematology and Oncology·Wolfgang SchwingerChristian Urban
Jun 2, 2015·Bone Marrow Transplantation·K K BallenJ Kurtzberg
Oct 1, 2014·Pain Management Nursing : Official Journal of the American Society of Pain Management Nurses·Kelly VasquenzaSabine Kost-Byerly
Jun 15, 2011·Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation·Jacob RozmusFrederick D Goldman
Jun 30, 2004·The Annals of Thoracic Surgery·Jann-Yuan WangYung-Chie Lee
Jun 9, 2015·Molecular Therapy. Methods & Clinical Development·Daniel C MendezPhilip H Schwartz
Aug 24, 2012·Journal of Child Neurology·Agnese SuppiejMaurizio Scarpa
Jun 24, 2017·Genetics in Medicine : Official Journal of the American College of Medical Genetics·Linda A BradleyGlenn E Palomaki
May 10, 2011·Bone Marrow Transplantation·M MynarekK-W Sykora
Nov 1, 2011·Expert Review of Endocrinology & Metabolism·Lorne A Clarke
Jun 15, 2011·Expert Review of Hematology·Jacob Rozmus, Kirk R Schultz
Feb 23, 2020·Current Stem Cell Research & Therapy·Ifrah AnwarZeeshan Shaukat
Nov 22, 2017·Expert Opinion on Orphan Drugs·Molly StapletonShunji Tomatsu
Jun 13, 2020·Journal of Molecular Medicine : Official Organ of the Gesellschaft Deutscher Naturforscher Und Ärzte·Andrés Felipe LealCarlos Javier Alméciga-Díaz
May 1, 2020·Frontiers in Molecular Biosciences·Jacob M FavretDaesung Shin
Aug 15, 2019·British Journal of Haematology·Maria GabelliRobert Chiesa
May 19, 2019·Transplant Infectious Disease : an Official Journal of the Transplantation Society·Eman F BoteinAli M Shaltot
Dec 12, 2020·Frontiers in Molecular Biosciences·Mariola J Edelmann, Gustavo H B Maegawa
Jun 23, 2018·Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation·Heather AlleweltJoanne Kurtzberg

❮ Previous
Next ❯

Related Concepts

Related Feeds

Blood Brain Barrier

The blood brain barrier is a border that separates blood from cerebrospinal fluid. Discover the latest search on this highly selective semipermeable membrane here.

Blood Brain Barrier Regulation in Health & Disease

The blood brain barrier is essential in regulating the movement of molecules and substances in and out of the brain. Disruption to the blood brain barrier and changes in permeability allow pathogens and inflammatory molecules to cross the barrier and may play a part in the pathogenesis of neurodegenerative disorders. Here is the latest research in this field.

Blood Brain Barrier Chips

The blood brain barrier (BBB) is comprised of endothelial cells that regulate the influx and outflux of plasma concentrations. Lab-on-a-chip devices allow scientists to model diseases and mechanisms such as the passage of therapeutic antibodies across the BBB. Discover the latest research on BBB chips here.

Blood And Marrow Transplantation

The use of hematopoietic stem cell transplantation or blood and marrow transplantation (bmt) is on the increase worldwide. BMT is used to replace damaged or destroyed bone marrow with healthy bone marrow stem cells. Here is the latest research on bone and marrow transplantation.

Related Papers

Seminars in Hematology
Vinod K Prasad, Joanne Kurtzberg
Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation
Jaap Jan BoelensEUROCORD, Inborn error Working Party of EBMT and Duke University
© 2022 Meta ULC. All rights reserved