AAV gene therapy for mucopolysaccharidoses

被引：0

作者：

Khan, Shaukat ^{[1
]}

Ago, Yasuhiko ^{[1
]}

Tomatsu, Shunji ^{[1
,2
,3
,4
]}

机构：

[1] Nemours Childrens Hlth, Wilmington, DE 19803 USA

[2] Univ Delaware, Fac Arts & Sci, Newark, DE 19716 USA

[3] Gifu Univ, Grad Sch Med, Dept Pediat, Gifu, Japan

[4] Thomas Jefferson Univ, Dept Pediat, Philadelphia, PA 19107 USA

来源：

MED | 2025年 / 6卷 / 04期

关键词：

D O I：

10.1016/j.medj.2025.100609

中图分类号：

R-3 [医学研究方法]; R3 [基础医学];

学科分类号：

1001 ;

摘要：

Adeno-associated viral (AAV) vectors emerge as a promising treatment option for genetic diseases. They recognized as the most effective in vivo gene therapy due to their safety, efficacy, and ability to provide term therapeutic transgene expression.1-3Rossi et al.1 have demonstrated that AAV gene therapy is effective and safe for treating patients with mucopolysaccharidosis VI, paving the way for potential treatments copolysaccharidoses.

引用

页数：3

共 10 条

[1]

Bigger BW, 2014, DISCOV MED, V17, P207

[2] The Bespoke Gene Therapy Consortium: facilitating development of AAV gene therapies for rare diseases [J].

Brooks, P. J. ;

Miller, Timothy M. ;

Revah, Frederic ;

Suh, Junghae ;

Garrison, Bradley R. ;

Starke, Lawrence C. ;

MacLachlan, Timothy K. ;

Neilan, Edward G. ;

Raychaudhuri, Gopa ;

Kassim, Sadik H. ;

Dehdashti, Jean ;

Rutter, Joni L. .

NATURE REVIEWS DRUG DISCOVERY, 2024, 23 (03) :157-158

[3]

Brunetti-Pierri N, 2022, NEJM EVID, V1, DOI [10.1056/evidoa2200052, 10.1056/EVIDoa2200052]

[4]

Clarke Lorne A., 2008, Expert Reviews in Molecular Medicine, V10, P1, DOI 10.1017/S1462399408000550

[5] Current Status on Clinical Development of Adeno-Associated Virus-Mediated Liver-Directed Gene Therapy for Inborn Errors of Metabolism [J].

Ginocchio, Virginia Maria ;

Ferla, Rita ;

Auricchio, Alberto ;

Brunetti-Pierri, Nicola .

HUMAN GENE THERAPY, 2019, 30 (10) :1204-1210

[6] Preclinical evaluation of FLT190, a liver-directed AAV gene therapy for Fabry disease [J].

Jeyakumar, Jey M. ;

Kia, Azadeh ;

Tam, Lawrence C. S. ;

McIntosh, Jenny ;

Spiewak, Justyna ;

Mills, Kevin ;

Heywood, Wendy ;

Chisari, Elisa ;

Castaldo, Noemi ;

Verhoef, Daniel ;

Hosseini, Paniz ;

Kalcheva, Petya ;

Cocita, Clement ;

Miranda, Carlos J. ;

Canavese, Miriam ;

Khinder, Jaminder ;

Rosales, Cecilia ;

Hughes, Derralynn ;

Sheridan, Rose ;

Corbau, Romuald ;

Nathwani, Amit .

GENE THERAPY, 2023, 30 (06) :487-502

[7] Adenovirus-Associated Virus Vector-Mediated Gene Transfer in Hemophilia B [J].

Nathwani, Amit C. ;

Tuddenham, Edward G. D. ;

Rangarajan, Savita ;

Rosales, Cecilia ;

McIntosh, Jenny ;

Linch, David C. ;

Chowdary, Pratima ;

Riddell, Anne ;

Pie, Arnulfo Jaquilmac ;

Harrington, Chris ;

O'Beirne, James ;

Smith, Keith ;

Pasi, John ;

Glader, Bertil ;

Rustagi, Pradip ;

Ng, Catherine Y. C. ;

Kay, Mark A. ;

Zhou, Junfang ;

Spence, Yunyu ;

Morton, Christopher L. ;

Allay, James ;

Coleman, John ;

Sleep, Susan ;

Cunningham, John M. ;

Srivastava, Deokumar ;

Basner-Tschakarjan, Etiena ;

Mingozzi, Federico ;

High, Katherine A. ;

Gray, John T. ;

Reiss, Ulrike M. ;

Nienhuis, Arthur W. ;

Davidoff, Andrew M. .

NEW ENGLAND JOURNAL OF MEDICINE, 2011, 365 (25) :2357-2365

[8]

Rossi A., 2024, Med, V6

[9] Hematopoietic stem cell transplantation improves the high incidence of neutralizing allo-antibodies observed in Hurler's syndrome after pharmacological enzyme replacement therapy [J].

Saif, Muhammad Ameer ;

Bigger, Brian W. ;

Brookes, Karen E. ;

Mercer, Jean ;

Tylee, Karen L. ;

Church, Heather J. ;

Bonney, Denise K. ;

Jones, Simon ;

Wraith, J. Ed ;

Wynn, Robert F. .

HAEMATOLOGICA-THE HEMATOLOGY JOURNAL, 2012, 97 (09) :1320-1328

[10]

Wang R, 2022, MOL GENET METAB, V135, pS126, DOI 10.1016/j.ymgme.2021.11.335

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