Maria Luisa Jaén

Long-Term Efficacy and Safety of Insulin and Glucokinase Gene Therapy for Diabetes: 8-Year Follow-Up in Dogs

Maria Luisa Jaén, Laia Vilà, Ivet Elias et al. · Molecular Therapy — Methods & Clinical Development · 2017 · 50 citations · Full text

Medicine, Molecular Medicine, Veterinary Clinical Sciences +12

Treatment of skeletal and non-skeletal alterations of Mucopolysaccharidosis type IVA by AAV-mediated gene therapy

Joan Bertolin, Víctor Sánchez, Albert Ribera et al. · Nature Communications · 2021 · 38 citations · Full text

Medicine, Molecular Genetics, Pathology +17

Seven-year follow-up of durability and safety of AAV CNS gene therapy for a lysosomal storage disorder in a large animal

Sara Marcó, Virginia Haurigot, Maria Luisa Jaén et al. · Molecular Therapy — Methods & Clinical Development · 2021 · 34 citations · Full text

Medicine, Neurology, Neuroscience +17

Treatment of infantile-onset Pompe disease in a rat model with muscle-directed AAV gene therapy

Sergio Muñoz, Joan Bertolin, Verónica Jiménez et al. · Molecular Metabolism · 2024 · 17 citations · Full text

Medicine, Genetics, Molecular Biology +16

Reversion of metabolic dysfunction-associated steatohepatitis by skeletal muscle-directed FGF21 gene therapy

Verónica Jiménez, Victor Sacristan, Claudia Jambrina et al. · Molecular Therapy · 2024 · 16 citations · Full text

Medicine, Biochemistry, Molecular Medicine +17