Search Result "mucopolysaccharidosis"


Immunology of Neonatal Gene Transfer

Journal: Current Gene Therapy
Volume: 7 Issue: 5 Year: 2007 Page: 403-410
Author(s): Katherine P. Ponder

Review Article

Genetics and Gene Therapy in Hunter Disease

Journal: Current Gene Therapy
Volume: 18 Issue: 2 Year: 2018 Page: 90-95
Author(s): S. Sestito,F. Falvo,C. Scozzafava,R. Apa,L. Pensabene,G. Bonapace,M.T. Moricca,D. Concolino

Case Report

Bone Remodeling in an Mps-1h Girl after Hematopoietic Stem CellTransplantation along with Enzymatic Replacement Therapy

Journal: Endocrine, Metabolic & Immune Disorders - Drug Targets
Volume: 22 Issue: 14 Year: 2022 Page: 1425-1432
Author(s): Maria Felicia Faienza

Targeting Brain Disease in Mucopolysaccharidoses

Ebook: Frontiers in Nanomedicine

Volume: 2 Year: 2017
Author(s): Marika Salvalaio,Laura Rigon,Francesca D’Avanzo,Elisa Legnini,Valeria Balmaceda Valdez,Alessandra Zanetti,Rosella Tomanin
Doi: 10.2174/9781681084930117020009

Therapies for Neurological Disease in the Mucopolysaccharidoses

Journal: Current Gene Therapy
Volume: 11 Issue: 2 Year: 2011 Page: 132-143
Author(s): Donald S. Anson, Chantelle McIntyre, Sharon Byers

Treatment of Lysosomal Storage Diseases: Recent Patents and Future Strategies

Journal: Recent Patents on Endocrine, Metabolic & Immune Drug Discovery
Volume: 8 Issue: 1 Year: 2014 Page: 9-25
Author(s): Saida Ortolano,Irene Vieitez,Carmen Navarro,Carlos Spuch

Expression of Active α-N-Acetylglucosaminidase/TAT Chimerae in Cultured Spodoptera frugiperda Cells

Journal: Protein & Peptide Letters
Volume: 13 Issue: 4 Year: 2006 Page: 353-356
Author(s): Judith C. Bandsmer, Tessa N. Campbell, Ian Cheyne, Francis Y.M. Choy

Perinatal Gene Transfer to the Liver

Journal: Current Pharmaceutical Design
Volume: 17 Issue: 2 Year: 2011 Page: 2528-2541
Author(s): Tristan R. McKay, Ahad A. Rahim, Suzanne M.K. Buckley, Natalie J. Ward, Jerry K.Y.Chan, Steven J. Howe, Simon N. Waddington

Mini-Review Article

Clinical Development of Cell Therapies to Halt Lysosomal Storage Diseases:Results and Lessons Learned

Journal: Current Gene Therapy
Volume: 22 Issue: 3 Year: 2022 Page: 191-213
Author(s): Valeria Graceffa

Gene Therapy for Lysosomal Storage Diseases: Progress, Challenges and Future Prospects

Journal: Current Pharmaceutical Design
Volume: 17 Issue: 2 Year: 2011 Page: 2558-2574
Author(s): Sergey S. Seregin, Andrea Amalfitano

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