Gene Therapy for Neuronopathic Mucopolysaccharidoses: State of the Art

Gene Therapy for Neuronopathic Mucopolysaccharidoses: State of the Art

The need for long-lasting and transformative therapies for mucopolysaccharidoses (MPS) cannot be understated. Currently, many forms of MPS lack a specific treatment and in other cases available therapies, such as enzyme replacement therapy (ERT), do not reach important areas such as the central nerv...

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Bibliographic Details
Main Authors: María José de Castro, Mireia del Toro, Roberto Giugliani, María Luz Couce
Format: Article
Language:English
Published: MDPI AG 2021-08-01
Series:International Journal of Molecular Sciences
Subjects:
mucopolysaccharidoses
gene therapy
viral vectors
adeno-associated virus
lentivirus
central nervous system
Online Access:https://www.mdpi.com/1422-0067/22/17/9200

Internet

https://www.mdpi.com/1422-0067/22/17/9200

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