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Literature DB >> 23449107

Gene therapy for Canavan's disease takes a step forward.

Seemin S Ahmed¹, Guangping Gao.

Abstract

Entities: Disease

Mesh：

Substances：

Year: 2013 PMID： 23449107 PMCID： PMC3589154 DOI： 10.1038/mt.2013.25

Source DB: PubMed Journal: Mol Ther ISSN： 1525-0016 Impact factor: 11.454

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12 in total

Review 1. Molecular basis of Canavan disease.

Authors: R Matalon; K Michals-Matalon
Journal: Eur J Paediatr Neurol Date: 1998 Impact factor: 3.140

2. Prenatal diagnosis for Canavan disease: the use of DNA markers.

Authors: R Matalon; R Kaul; G P Gao; K Michals; R G Gray; S Bennett-Briton; A Norman; M Smith; C Jakobs
Journal: J Inherit Metab Dis Date: 1995 Impact factor: 4.982

3. Aspartoacylase deficiency and N-acetylaspartic aciduria in patients with Canavan disease.

Authors: R Matalon; K Michals; D Sebesta; M Deanching; P Gashkoff; J Casanova
Journal: Am J Med Genet Date: 1988-02

4. Aspartoacylase gene transfer to the mammalian central nervous system with therapeutic implications for Canavan disease.

Authors: P Leone; C G Janson; L Bilaniuk; Z Wang; F Sorgi; L Huang; R Matalon; R Kaul; Z Zeng; A Freese; S W McPhee; E Mee; M J During; L Bilianuk
Journal: Ann Neurol Date: 2000-07 Impact factor: 10.422

5. Restoration of aspartoacylase activity in CNS neurons does not ameliorate motor deficits and demyelination in a model of Canavan disease.

Authors: Matthias Klugmann; Claudia B Leichtlein; C Wymond Symes; Tadao Serikawa; Deborah Young; Matthew J During
Journal: Mol Ther Date: 2005-05 Impact factor: 11.454

6. Clinical protocol. Gene therapy of Canavan disease: AAV-2 vector for neurosurgical delivery of aspartoacylase gene (ASPA) to the human brain.

Authors: Christopher Janson; Scott McPhee; Larissa Bilaniuk; John Haselgrove; Mark Testaiuti; Andrew Freese; Dah-Jyuu Wang; David Shera; Peter Hurh; Joan Rupin; Elizabeth Saslow; Olga Goldfarb; Michael Goldberg; Ghassem Larijani; William Sharrar; Larisa Liouterman; Angelique Camp; Edwin Kolodny; Jude Samulski; Paola Leone
Journal: Hum Gene Ther Date: 2002-07-20 Impact factor: 5.695

7. Long-term follow-up after gene therapy for canavan disease.

Authors: Paola Leone; David Shera; Scott W J McPhee; Jeremy S Francis; Edwin H Kolodny; Larissa T Bilaniuk; Dah-Jyuu Wang; Mitra Assadi; Olga Goldfarb; H Warren Goldman; Andrew Freese; Deborah Young; Matthew J During; R Jude Samulski; Christopher G Janson
Journal: Sci Transl Med Date: 2012-12-19 Impact factor: 17.956

Gene therapy for Canavan's disease takes a step forward.

Review 1. Molecular basis of Canavan disease.

2. Prenatal diagnosis for Canavan disease: the use of DNA markers.

3. Aspartoacylase deficiency and N-acetylaspartic aciduria in patients with Canavan disease.

4. Aspartoacylase gene transfer to the mammalian central nervous system with therapeutic implications for Canavan disease.

5. Restoration of aspartoacylase activity in CNS neurons does not ameliorate motor deficits and demyelination in a model of Canavan disease.

6. Clinical protocol. Gene therapy of Canavan disease: AAV-2 vector for neurosurgical delivery of aspartoacylase gene (ASPA) to the human brain.

7. Long-term follow-up after gene therapy for canavan disease.

8. Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes.

9. Human Gene Mutation Database (HGMD): 2003 update.

10. Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN.

Review 1. Canavan Disease as a Model for Gene Therapy-Mediated Myelin Repair.