Literature DB >> 18545253

The controversial and changing role of haematopoietic cell transplantation for lysosomal storage disorders: an update.

A M Rovelli1.   

Abstract

Haematopoietic cell transplantation (HCT) can benefit some selected subsets of patients with lysosomal diseases. Results had been impressive in children with MPS I-H, but poor in other disorders. Careful, multidisciplinary decision-making regarding whether to recommend HCT and how to provide optimal pre- and post-HCT care has proven essential to increase the likelihood of a good outcome.

Entities:  

Mesh：

Year:  2008        PMID： 18545253     DOI： 10.1038/bmt.2008.62

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


  18 in total

1.  Clinical outcomes following hematopoietic stem cell transplantation for the treatment of mucopolysaccharidosis VI.

Authors:  Sean Turbeville; Helen Nicely; J Douglas Rizzo; Tanya L Pedersen; Paul J Orchard; Mitchell E Horwitz; Edwin M Horwitz; Paul Veys; Carmem Bonfim; Amal Al-Seraihy
Journal:  Mol Genet Metab       Date:  2010-10-25       Impact factor: 4.797

Review 2.  Gene therapy for the neurological manifestations in lysosomal storage disorders.

Authors:  Seng H Cheng
Journal:  J Lipid Res       Date:  2014-03-29       Impact factor: 5.922

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.  Course and management of allogeneic stem cell transplantation in patients with mitochondrial neurogastrointestinal encephalomyopathy.

Authors:  Massimiliano Filosto; Mauro Scarpelli; Paola Tonin; Giovanna Lucchini; Fabio Pavan; Francesca Santus; Rossella Parini; Maria Alice Donati; Maria Sofia Cotelli; Valentina Vielmi; Alice Todeschini; Francesco Canonico; Giuliano Tomelleri; Alessandro Padovani; Attilio Rovelli
Journal:  J Neurol       Date:  2012-06-19       Impact factor: 4.849

Review 5.  Therapies for the bone in mucopolysaccharidoses.

Authors:  Shunji Tomatsu; Carlos J Alméciga-Díaz; Adriana M Montaño; Hiromasa Yabe; Akemi Tanaka; Vu Chi Dung; Roberto Giugliani; Francyne Kubaski; Robert W Mason; Eriko Yasuda; Kazuki Sawamoto; William Mackenzie; Yasuyuki Suzuki; Kenji E Orii; Luis A Barrera; William S Sly; Tadao Orii
Journal:  Mol Genet Metab       Date:  2014-12-09       Impact factor: 4.797

Review 6.  Therapeutic liver repopulation for phenylketonuria.

Authors:  Cary O Harding; K M Gibson
Journal:  J Inherit Metab Dis       Date:  2010-05-22       Impact factor: 4.982

Review 7.  Mucopolysaccharidosis VI.

Authors:  Vassili Valayannopoulos; Helen Nicely; Paul Harmatz; Sean Turbeville
Journal:  Orphanet J Rare Dis       Date:  2010-04-12       Impact factor: 4.123

Review 8.  Recent gene therapy advancements for neurological diseases.

Authors:  Sahana Nagabhushan Kalburgi; Nadia N Khan; Steven J Gray
Journal:  Discov Med       Date:  2013-02       Impact factor: 2.970

9.  Neonatal bone marrow transplantation prevents bone pathology in a mouse model of mucopolysaccharidosis type I.

Authors:  Alice Pievani; Isabella Azario; Laura Antolini; Tsutomu Shimada; Pravin Patel; Cristina Remoli; Benedetta Rambaldi; Maria Grazia Valsecchi; Mara Riminucci; Andrea Biondi; Shunji Tomatsu; Marta Serafini
Journal:  Blood       Date:  2014-10-08       Impact factor: 22.113

Review 10.  Anaesthesia and airway management in mucopolysaccharidosis.

Authors:  Robert Walker; Kumar G Belani; Elizabeth A Braunlin; Iain A Bruce; Henrik Hack; Paul R Harmatz; Simon Jones; Richard Rowe; Guirish A Solanki; Barbara Valdemarsson
Journal:  J Inherit Metab Dis       Date:  2012-11-30       Impact factor: 4.982
View more

北京卡尤迪生物科技股份有限公司 © 2022-2023.