Adeno-associated viral gene therapy for mucopolysaccharidoses exhibiting neurodegeneration

被引：0
|
作者：
Adeline A. Lau
Kim M. Hemsley
机构：
[1] South Australian Health and Medical Research Institute (SAHMRI),Lysosomal Diseases Research Unit, Nutrition and Metabolism Theme
来源：
Journal of Molecular Medicine | 2017年 / 95卷
关键词：
Gene therapy; Viral vectors; Lysosomal storage disease; Mucopolysaccharidosis; Central nervous system;
D O I：
暂无
中图分类号：
学科分类号：
摘要：
The mucopolysaccharidoses (MPS) are a subgroup of lysosomal storage disorders that are caused by mutations in the genes involved in glycosaminoglycan breakdown. Multiple organs and tissues are affected, including the central nervous system. At present, hematopoietic stem cell transplantation and enzyme replacement therapies are approved for some of the (non-neurological) MPS. Treatments that effectively ameliorate the neurological aspects of the disease are being assessed in clinical trials. This review will focus on the recent outcomes and planned viral vector-mediated gene therapy clinical trials, and the pre-clinical data that supported these studies, for MPS-I (Hurler/Scheie syndrome), MPS-II (Hunter syndrome), and MPS-IIIA and -IIIB (Sanfilippo syndrome).
引用
收藏
页码：1043 / 1052
页数：9
相关论文
共 50 条
  • [31] Adeno-associated viral vectors
    Lehtonen, E
    Tenenbaum, L
    VIRAL VECTORS FOR TREATING DISEASES OF THE NERVOUS SYSTEM, 2003, 55 : 65 - 98
  • [32] My Pathway to Adeno-Associated Virus and Adeno-Associated Virus Gene Therapy: A Personal Perspective
    Carter, Barrie J.
    HUMAN GENE THERAPY, 2020, 31 (9-10) : 494 - 498
  • [33] Gene therapy for Parkinson's disease using recombinant adeno-associated viral vectors
    Muramatsu, S
    Tsukada, H
    Nakano, I
    Ozawa, K
    EXPERT OPINION ON BIOLOGICAL THERAPY, 2005, 5 (05) : 663 - 671
  • [34] Development Of Recombinant Adeno-Associated Viral Vector (raav) Gene Therapy For Cystic Fibrosis
    Carlon, M. S.
    Vidovic, D.
    Ensinck, M.
    Da Cunha, M. F.
    Dekkers, J. F.
    Bijvelds, M.
    De Jonge, H.
    Ramalho, A. S.
    De Boeck, K.
    Sermet-Gaudelus, I.
    Gijsbers, R.
    Edelman, A.
    Beekman, J.
    Debyser, Z.
    AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE, 2017, 195
  • [35] An Adeno-Associated Viral Vector Capable of Penetrating the Mucus Barrier to Inhaled Gene Therapy
    Duncan, Gregg A.
    Kim, Namho
    Colon-Cortes, Yanerys
    Rodriguez, Jason
    Mazur, Marina
    Birket, Susan E.
    Rowe, Steven M.
    West, Natalie E.
    Livraghi-Butrico, Alessandra
    Boucher, Richard C.
    Hanes, Justin
    Aslanidi, George
    Suk, Jung Soo
    MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT, 2018, 9 : 296 - 304
  • [36] Development and Validation of a Novel Adeno-Associated Viral Gene Therapy for Mucopolysaccharidosis IIIB (MPSIIIB)
    Piguet, Francoise
    MOLECULAR THERAPY, 2023, 31 (04) : 651 - 651
  • [37] Adeno-associated viral vectors for in utero gene therapy of inherited metabolic disease.
    Patel, S
    Gau, CL
    Cederbaum, S
    Busuttil, RW
    Lipshutz, GS
    JOURNAL OF INVESTIGATIVE MEDICINE, 2006, 54 (01) : S146 - S146
  • [38] Development and validation of a novel adeno-associated viral gene therapy for Mucopolysaccharidosis IIIB (MPSIIIB)
    Ballout, N.
    Banchi, E. G.
    Audouard, E.
    Alonso, R.
    Verdu, L.
    Santiago, C.
    Rouahi, M.
    de Saint Denis, T.
    Ausseil, J.
    Piguet, F.
    HUMAN GENE THERAPY, 2024, 35 (3-4) : A7 - A7
  • [39] The Application of Adeno-Associated Viral Vector Gene Therapy to the Treatment of Fragile X Syndrome
    Hampson, David R.
    Hooper, Alexander W. M.
    Niibori, Yosuke
    BRAIN SCIENCES, 2019, 9 (02)
  • [40] Membrane chromatography purification of adenovirus and adeno-associated viral vectors for gene therapy.
    Lajmi, A
    ABSTRACTS OF PAPERS OF THE AMERICAN CHEMICAL SOCIETY, 2005, 229 : U187 - U187
12345