Development of an Antisense Oligonucleotide-Mediated Exon Skipping Therapeutic Strategy for Mucolipidosis II: Validation at RNA Level-Reference-Cited by-同舟云学术

Development of an Antisense Oligonucleotide-Mediated Exon Skipping Therapeutic Strategy for Mucolipidosis II: Validation at RNA Level

Published:2020-07-01 Issue:13-14 Volume:31 Page:775-783
ISSN:1043-0342
Container-title:Human Gene Therapy
language:en
Short-container-title:Human Gene Therapy

Author:

Matos Liliana¹²,Vilela Regina¹,Rocha Melissa¹,Santos Juliana I.¹³,Coutinho Maria Francisca¹²,Gaspar Paulo⁴,Prata Maria João³⁵,Alves Sandra¹²

Affiliation:

1. Research and Development Unit, Department of Human Genetics, National Health Institute Doutor Ricardo Jorge, Porto, Portugal.

2. Center for the Study of Animal Science, CECA-ICETA, University of Porto, Porto, Portugal.

3. Biology Department, Faculty of Sciences, University of Porto, Porto, Portugal.

4. Newborn Screening, Metabolism and Genetics Unit, Department of Human Genetics, National Health Institute Doutor Ricardo Jorge, Porto, Portugal.

5. i3S, Institute of Research and Innovation in Health/IPATIMUP, Institute of Molecular Pathology and Immunology of the University of Porto, Porto, Portugal.

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

https://www.liebertpub.com/doi/pdf/10.1089/hum.2020.034

Reference38 articles.

1. FDA Approves Eteplirsen for Duchenne Muscular Dystrophy: The Next Chapter in the Eteplirsen Saga

2. A Half-Century History of Applications of Antisense Oligonucleotides in Medicine, Agriculture and Forestry: We Should Continue the Journey

3. Antisense oligonucleotides in therapy for neurodegenerative disorders

4. Mannose phosphorylation in health and disease

5. Mannose-6-phosphate pathway: A review on its role in lysosomal function and dysfunction

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