Development of an antisense oligonucleotides-based therapy for mucolipidosis type II α/β: in vivo studies

Introduction: The RNA molecule has become an increasingly promising target for the therapy of various diseases. Mucolipidosis type II α/β (ML II α/β) is one of the most severe Lysosomal Storage Disorders and is caused by the deficiency of the enzyme GlcNAc-1- phosphotransferase. This enzyme is respo...

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Bibliographic Details
Main Author: Gonçalves, Mariana (author)
Other Authors: Matos, Liliana (author), Santos, Juliana Inês (author), Coutinho, Maria Francisca (author), Prata, Maria João (author), Pires, Maria João (author), Oliveira, Paula (author), Alves, Sandra (author)
Format: conferenceObject
Language:eng
Published: 2022
Subjects:
Doenças Genéticas
Genética Humana
Doenças Lisossomais de sobrecarga
Mucolipidose tipo II
Online Access:http://hdl.handle.net/10400.18/8191
Country:Portugal
Oai:oai:repositorio.insa.pt:10400.18/8191

Internet

http://hdl.handle.net/10400.18/8191

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