Literature DB >> 10518291

Bone marrow transplantation in mucopolysaccharidosis type IIIA: a comparison of an early treated patient with his untreated sibling.

P Sivakumur1, J E Wraith.   

Abstract

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Year:  1999        PMID： 10518291     DOI： 10.1023/a:1005526628598

Source DB:  PubMed          Journal:  J Inherit Metab Dis        ISSN： 0141-8955            Impact factor:   4.982


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  2 in total

1.  Allogeneic bone marrow transplantation for lysosomal storage diseases. The European Group for Bone Marrow Transplantation.

Authors:  P M Hoogerbrugge; O F Brouwer; P Bordigoni; O Ringden; P Kapaun; J J Ortega; A O'Meara; G Cornu; G Souillet; D Frappaz
Journal:  Lancet       Date:  1995-06-03       Impact factor: 79.321

Review 2.  Management of mucopolysaccharidosis type III.

Authors:  M A Cleary; J E Wraith
Journal:  Arch Dis Child       Date:  1993-09       Impact factor: 3.791
  2 in total
  22 in total

1.  Liver production of sulfamidase reverses peripheral and ameliorates CNS pathology in mucopolysaccharidosis IIIA mice.

Authors:  Albert Ruzo; Miquel Garcia; Albert Ribera; Pilar Villacampa; Virginia Haurigot; Sara Marcó; Eduard Ayuso; Xavier M Anguela; Carles Roca; Judith Agudo; David Ramos; Jesús Ruberte; Fatima Bosch
Journal:  Mol Ther       Date:  2011-10-18       Impact factor: 11.454

2.  Delivery of an enzyme-IGFII fusion protein to the mouse brain is therapeutic for mucopolysaccharidosis type IIIB.

Authors:  Shih-Hsin Kan; Mika Aoyagi-Scharber; Steven Q Le; Jon Vincelette; Kazuhiro Ohmi; Sherry Bullens; Daniel J Wendt; Terri M Christianson; Pascale M N Tiger; Jillian R Brown; Roger Lawrence; Bryan K Yip; John Holtzinger; Anil Bagri; Danielle Crippen-Harmon; Kristen N Vondrak; Zhi Chen; Chuck M Hague; Josh C Woloszynek; Diana S Cheung; Katherine A Webster; Evan G Adintori; Melanie J Lo; Wesley Wong; Paul A Fitzpatrick; Jonathan H LeBowitz; Brett E Crawford; Stuart Bunting; Patricia I Dickson; Elizabeth F Neufeld
Journal:  Proc Natl Acad Sci U S A       Date:  2014-09-29       Impact factor: 11.205

3.  Neonatal Bone Marrow Transplantation in MPS IIIA Mice.

Authors:  Adeline A Lau; N Jannah Shamsani; Leanne K Winner; Sofia Hassiotis; Barbara M King; John J Hopwood; Kim M Hemsley
Journal:  JIMD Rep       Date:  2012-08-10

Review 4.  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

5.  Early Umbilical Cord Blood-Derived Stem Cell Transplantation Does Not Prevent Neurological Deterioration in Mucopolysaccharidosis Type III.

Authors:  Lindsey Welling; Jan Pieter Marchal; Peter van Hasselt; Ans T van der Ploeg; Frits A Wijburg; Jaap Jan Boelens
Journal:  JIMD Rep       Date:  2014-09-26

6.  Caprine mucopolysaccharidosis IIID: a preliminary trial of enzyme replacement therapy.

Authors:  E Downs-Kelly; M Z Jones; J Alroy; K T Cavanagh; B King; R E Lucas; J C Baker; S A Kraemer; J J Hopwood
Journal:  J Mol Neurosci       Date:  2000-12       Impact factor: 3.444

7.  Hematopoietic stem cell and gene therapy corrects primary neuropathology and behavior in mucopolysaccharidosis IIIA mice.

Authors:  Alexander Langford-Smith; Fiona L Wilkinson; Kia J Langford-Smith; Rebecca J Holley; Ana Sergijenko; Steven J Howe; William R Bennett; Simon A Jones; Je Wraith; Catherine Lr Merry; Robert F Wynn; Brian W Bigger
Journal:  Mol Ther       Date:  2012-05-01       Impact factor: 11.454

8.  A Cure for Sanfilippo Syndrome? A Summary of Current Therapeutic Approaches and their Promise.

Authors:  Yewande Pearse; Michelina Iacovino
Journal:  Med Res Arch       Date:  2020-02-21

Review 9.  Allogeneic stem cell transplantation for the treatment of lysosomal and peroxisomal metabolic diseases.

Authors:  William Krivit
Journal:  Springer Semin Immunopathol       Date:  2004-09-25

Review 10.  Sanfilippo syndrome: a mini-review.

Authors:  M J Valstar; G J G Ruijter; O P van Diggelen; B J Poorthuis; F A Wijburg
Journal:  J Inherit Metab Dis       Date:  2008-04-04       Impact factor: 4.982
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