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Literature DB >> 16151419

Allogeneic BMT followed by substrate reduction therapy in a child with subacute Tay-Sachs disease.

J F M Jacobs, M A A P Willemsen, J J Groot-Loonen, R A Wevers, P M Hoogerbrugge.

Abstract

Entities: Disease Species

Mesh：

Substances：

Year: 2005 PMID： 16151419 DOI： 10.1038/sj.bmt.1705155

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

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14 in total

1. Substrate deprivation therapy in juvenile Sandhoff disease.

Authors: S B Wortmann; D J Lefeber; G Dekomien; M A A P Willemsen; R A Wevers; E Morava
Journal: J Inherit Metab Dis Date: 2009-11-04 Impact factor: 4.982

Review 2. The GM1 and GM2 Gangliosidoses: Natural History and Progress toward Therapy.

Authors: Debra S Regier; Richard L Proia; Alessandra D'Azzo; Cynthia J Tifft
Journal: Pediatr Endocrinol Rev Date: 2016-06

3. Substrate reduction therapy with miglustat in chronic GM2 gangliosidosis type Sandhoff: results of a 3-year follow-up.

Authors: Marcella Masciullo; Massimo Santoro; Anna Modoni; Enzo Ricci; Jerome Guitton; Pietro Tonali; Gabriella Silvestri
Journal: J Inherit Metab Dis Date: 2010-09-04 Impact factor: 4.982

4. Widespread correction of central nervous system disease after intracranial gene therapy in a feline model of Sandhoff disease.

Authors: V J McCurdy; H E Rockwell; J R Arthur; A M Bradbury; A K Johnson; A N Randle; B L Brunson; M Hwang; H L Gray-Edwards; N E Morrison; J A Johnson; H J Baker; N R Cox; T N Seyfried; M Sena-Esteves; D R Martin
Journal: Gene Ther Date: 2014-12-04 Impact factor: 5.250

Review 5. Therapeutic Strategies For Tay-Sachs Disease.

Authors: Jaqueline A Picache; Wei Zheng; Catherine Z Chen
Journal: Front Pharmacol Date: 2022-07-05 Impact factor: 5.988

6. Haematopoietic Stem Cell Transplantation Arrests the Progression of Neurodegenerative Disease in Late-Onset Tay-Sachs Disease.

Authors: Karolina M Stepien; Su Han Lum; J Edmond Wraith; Christian J Hendriksz; Heather J Church; David Priestman; Frances M Platt; Simon Jones; Ana Jovanovic; Robert Wynn
Journal: JIMD Rep Date: 2017-12-07

Allogeneic BMT followed by substrate reduction therapy in a child with subacute Tay-Sachs disease.

1. Substrate deprivation therapy in juvenile Sandhoff disease.

Review 2. The GM1 and GM2 Gangliosidoses: Natural History and Progress toward Therapy.

3. Substrate reduction therapy with miglustat in chronic GM2 gangliosidosis type Sandhoff: results of a 3-year follow-up.

4. Widespread correction of central nervous system disease after intracranial gene therapy in a feline model of Sandhoff disease.

Review 5. Therapeutic Strategies For Tay-Sachs Disease.

6. Haematopoietic Stem Cell Transplantation Arrests the Progression of Neurodegenerative Disease in Late-Onset Tay-Sachs Disease.

Review 7. Animal models of GM2 gangliosidosis: utility and limitations.

Review 8. New Approaches to Tay-Sachs Disease Therapy.

9. Genotype-phenotype correlation of gangliosidosis mutations using in silico tools and homology modeling.

10. Novel HEXA variants in Korean children with Tay-Sachs disease with regression of neurodevelopment from infancy.