Antisense Mediated Splicing Modulation For Inherited Metabolic Diseases: Challenges for Delivery-Reference-Cited by-同舟云学术

Antisense Mediated Splicing Modulation For Inherited Metabolic Diseases: Challenges for Delivery

Published:2014-02 Issue:1 Volume:24 Page:48-56
ISSN:2159-3337
Container-title:Nucleic Acid Therapeutics
language:en
Short-container-title:Nucleic Acid Therapeutics

Author:

Pérez Belen¹,Vilageliu Lluisa²,Grinberg Daniel²,Desviat Lourdes R.¹

Affiliation:

1. Centro de Biología Molecular Severo Ochoa, UAM-CSIC, Universidad Autónoma de Madrid, Madrid, Spain. Centro de Investigación Biomédica en Red de Enfermedades Raras, Madrid, Spain.

2. Departament de Genètica, Universitat de Barcelona, Institut de Biomedicina de la Universitat de Barcelona, Barcelona, Spain. Centro de Investigación Biomédica en Red de Enfermedades Raras, Barcelona, Spain.

Publisher

Mary Ann Liebert Inc

Subject

Drug Discovery,Genetics,Molecular Biology,Molecular Medicine,Biochemistry

Link

http://www.liebertpub.com/doi/pdf/10.1089/nat.2013.0453

Reference84 articles.

1. Antisense Oligonucleotide-Mediated Exon Skipping for Duchenne Muscular Dystrophy: Progress and Challenges

2. Phenylketonuria

3. Pseudoexon exclusion by antisense therapy in 6-pyruvoyl-tetrahydropterin synthase deficiency

4. The role of readthrough acetylcholinesterase in the pathophysiology of myasthenia gravis

5. Massively parallel sequencing, aCGH, and RNA-Seq technologies provide a comprehensive molecular diagnosis of Fanconi anemia

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