PROJECTS
Inherited metabolic diseases in animals
“Exploring in vivo U1 snRNA splicing modulation as an alternative therapy for MPS IIIC” Funding: National MPS Society - USA (2020-2021). PI. Liliana Matos
“Genetic Substrate Reduction Therapy for Mucopolysaccharidoses Toward a siRNA-containing nanoparticle targeted to brain cells” Funding: Sanfilippo Children’s Foundation (SFC) - Australia (2020-2021). PI. Francisca Coutinho
“Development of an antisense-mediated exon skipping approach as a therapeutic option for the ML II-causing mutation c.3503_3504delTC”. Funding: Asociación Nour de Mucolipidosis - Spain (2017-2020). PI. Sandra Alves
“Application of splicing approaches to exploit alternative therapies for Lysosomal Storage Diseases: in vitro and in vivo studies”. Fundação para a Ciência e Tecnologia. Ref. FCT/ PTDC /BBB-BMD/6301/2014. (2016-2019). PI. Sandra Alves
“Cellular models for the study of lysosomal dysfunction and correction mechanisms (CeMoLy)” Fundação para a Ciência e Tecnologia. Ref. FCT/ PTDC/BIM-MEC/4762/2014). (2016-2019). PI. Olga Amaral
“Genetic Substrate Reduction Therapy for Mucopolysaccharidoses Toward a siRNA-containing nanoparticle targeted to brain cells”. Bolsa SPDM de Apoio à Investigação Dr. Aguinaldo Cabral (2019-2020). PI. Francisca Coutinho.
European Joint Programme on Rare Diseases (EJP_RD). EJP RD 825575. EU Framework Programme Horizon 2020 (2019-2022). Sandra Alves is the representative of the National Institute of Health Dr. Ricardo Jorge (INSA) and of the Ministry of Health.
“Cost action CA17103 – Delivery of Antisense RNA Therapeutics”. EU Framework Programme Horizon 2020 (2018-2022). Sandra Alves is the national delegate in the Management Committee and Core Group Member and Liliana Matos is the substitute delegate.