Literature DB >> 18176609

Emerging trends in transplantation of inherited metabolic diseases.

V K Prasad1, J Kurtzberg.   

Abstract

Allogeneic hematopoietic stem cell transplantation (HSCT) can prolong life and improve its quality in patients with inherited metabolic diseases. HSCT offers a permanent source of enzyme replacement therapy and also might mediate nonhematopoietic cell regeneration or repair. Unrelated cord blood is an exciting newer graft source for treatment of patients with these fatal disorders, providing increased access to donors and significant clinical efficacy, particularly when transplantation is performed in early stages. Pre-transplant performance status is highly predictive of overall survival.

Entities:  

Mesh：

Year:  2008        PMID： 18176609     DOI： 10.1038/sj.bmt.1705970

Source DB:  PubMed          Journal:  Bone Marrow Transplant        ISSN： 0268-3369            Impact factor:   5.483


  25 in total

1.  Posttransplant autoimmune hemolytic anemia and other autoimmune cytopenias are increased in very young infants undergoing unrelated donor umbilical cord blood transplantation.

Authors:  Kristin M Page; Adam M Mendizabal; Vinod K Prasad; Paul L Martin; Suhag Parikh; Susan Wood; Gregory D Sempowski; Paul Szabolcs; Joanne Kurtzberg
Journal:  Biol Blood Marrow Transplant       Date:  2008-10       Impact factor: 5.742

2.  Partial Rescue of Biochemical Parameters After Hematopoietic Stem Cell Transplantation in a Patient with Prolidase Deficiency Due to Two Novel PEPD Mutations.

Authors:  Désirée Caselli; Rolando Cimaz; Roberta Besio; Antonio Rossi; Ersilia De Lorenzi; Raffaella Colombo; Luca Cantarini; Silvia Riva; Marco Spada; Antonella Forlino; Maurizio Aricò
Journal:  JIMD Rep       Date:  2011-09-27

Review 3.  Hematopoietic Stem Cell Transplantation for Mucopolysaccharidoses: Past, Present, and Future.

Authors:  Madeleine Taylor; Shaukat Khan; Molly Stapleton; Jianmin Wang; Jing Chen; Robert Wynn; Hiromasa Yabe; Yasutsugu Chinen; Jaap Jan Boelens; Robert W Mason; Francyne Kubaski; Dafne D G Horovitz; Anneliese L Barth; Marta Serafini; Maria Ester Bernardo; Hironori Kobayashi; Kenji E Orii; Yasuyuki Suzuki; Tadao Orii; Shunji Tomatsu
Journal:  Biol Blood Marrow Transplant       Date:  2019-02-14       Impact factor: 5.742

4.  Severe and fatal toxicity after hematopoietic stem cell transplantation in GNE defect-associated thrombocytopenia.

Authors:  Justyna Miśkiewicz-Bujna; Izabella Miśkiewicz-Migoń; Anna Panasiuk; Anna Kubica-Cielińska; Marzena Zielińska; Łukasz Waszczuk; Joanna Bladowska; Marcin Miś; Katarzyna Bąbol-Pokora; Wojciech Młynarski; Tomasz Ociepa; Krzysztof Kałwak; Ewa Gorczyńska; Marek Ussowicz
Journal:  Bone Marrow Transplant       Date:  2021-02-26       Impact factor: 5.483

5.  Therapeutic approaches for lysosomal storage diseases.

Authors:  Gregory M Pastores
Journal:  Ther Adv Endocrinol Metab       Date:  2010-08       Impact factor: 3.565

Review 6.  Cell replacement therapies: is it time to reprogram?

Authors:  Harald M Mikkers; Christian Freund; Christine L Mummery; Rob C Hoeben
Journal:  Hum Gene Ther       Date:  2014-09-17       Impact factor: 5.695

7.  Implication of cord blood for cell-based therapy in refractory childhood diseases.

Authors:  Young-Ho Lee
Journal:  Int J Stem Cells       Date:  2010-05       Impact factor: 2.500

Review 8.  Umbilical cord blood banking: an update.

Authors:  Merlin G Butler; Jay E Menitove
Journal:  J Assist Reprod Genet       Date:  2011-05-27       Impact factor: 3.412

Review 9.  Recent trends in mucopolysaccharidosis research.

Authors:  Hiroshi Kobayashi
Journal:  J Hum Genet       Date:  2018-11-19       Impact factor: 3.172

10.  Unrelated donor umbilical cord blood transplantation for inherited metabolic disorders in 159 pediatric patients from a single center: influence of cellular composition of the graft on transplantation outcomes.

Authors:  Vinod K Prasad; Adam Mendizabal; Suhag H Parikh; Paul Szabolcs; Timothy A Driscoll; Kristin Page; Sonali Lakshminarayanan; June Allison; Susan Wood; Deborah Semmel; Maria L Escolar; Paul L Martin; Shelly Carter; Joanne Kurtzberg
Journal:  Blood       Date:  2008-06-27       Impact factor: 22.113
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