Joan Bertolin

Universitat Autònoma de Barcelona — Spain

Medicine · Health Sciences

6h-index220citations6works16.02yr avg
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Research Topics

Lysosomal Storage Disorders Research(5), Trypanosoma species research and implications(3), Neurogenetic and Muscular Disorders Research(2), Autism Spectrum Disorder Research(1), Virus-based gene therapy research(1)

Publications6 total

Treatment of infantile-onset Pompe disease in a rat model with muscle-directed AAV gene therapy
Molecular Metabolism·2024· 16 citedOpen Access
Seven-year follow-up of durability and safety of AAV CNS gene therapy for a lysosomal storage disorder in a large animal
Molecular Therapy — Methods & Clinical Development·2021· 31 citedOpen Access
Treatment of skeletal and non-skeletal alterations of Mucopolysaccharidosis type IVA by AAV-mediated gene therapy
Nature Communications·2021· 35 citedOpen Access
Disease correction by AAV-mediated gene therapy in a new mouse model of mucopolysaccharidosis type IIID
Human Molecular Genetics·2017· 50 citedOpen Access
Progressive neurologic and somatic disease in a novel mouse model of human mucopolysaccharidosis type IIIC
Disease Models & Mechanisms·2016· 27 citedOpen Access
CNS-directed gene therapy for the treatment of neurologic and somatic mucopolysaccharidosis type II (Hunter syndrome)
JCI Insight·2016· 61 citedOpen Access

Frequent Co-authors

Miquel García(6), Fátima Bosch(6), Sara Marcó(5), Albert Ribera(5), Carles Roca(5), Xavier Sánchez(5), Víctor Sánchez(5), Jesús Ruberte(5), Virginia Haurigot(4), Xavier León(4), Anna Pujol(4), Sandra Motas(3), Maria Molas(3), Jennifer Pérez(3), Maria Luisa Jaén(3), Pilar Villacampa(2), Sergio Muñoz(2), Gemma Elias(2), Verónica Jiménez(2), Marc Navarro(2)