< Back to previous page
Project
HERCULES: Development of an innovative muscle-directed gene therapy platform for lysosomal storage disorders (FWOAL1073)
Pompe’s disease (GSD II) is one of the most common hereditary
muscle disorders that is caused by a genetic defect in acid
glucosidase (GAA). Typically, the afflicted patients gradually lose all
muscle function and ultimately die from heart and lung failure.
Current treatment with recombinant proteins alleviates some of the
symptoms but are not curative and largely inadequate since the large
majority of patients ultimately still die from their underlying genetic
disorder. Hence, there is an urgent need to develop an efficacious
and safe therapy that tackles the genetic cause of the disease. This
proposal’s overall vision is to develop the next-generation muscledirected gene therapy to effectively treat patients suffering from lifethreatening hereditary muscle disorders. To realize this vision, the
HERCULES proposal’s overarching significant goals are: (i) to
develop a unique and novel gene therapy technology specifically
designed to increase the delivery and production of the therapeutic
genes in the affected target tissues; (ii) to validate the efficacy and
safety of this next-generation gene therapy platform in a preclinical
animal model for Pompe disease. This HERCULES proposal is highly
focused and may ultimately pave the way towards a phase I clinical
trial in patients suffering from Pompe disease, which highlights its
translational potential and medical relevance with possibly broad
implications for patients suffering from other rare muscle disorders.
muscle disorders that is caused by a genetic defect in acid
glucosidase (GAA). Typically, the afflicted patients gradually lose all
muscle function and ultimately die from heart and lung failure.
Current treatment with recombinant proteins alleviates some of the
symptoms but are not curative and largely inadequate since the large
majority of patients ultimately still die from their underlying genetic
disorder. Hence, there is an urgent need to develop an efficacious
and safe therapy that tackles the genetic cause of the disease. This
proposal’s overall vision is to develop the next-generation muscledirected gene therapy to effectively treat patients suffering from lifethreatening hereditary muscle disorders. To realize this vision, the
HERCULES proposal’s overarching significant goals are: (i) to
develop a unique and novel gene therapy technology specifically
designed to increase the delivery and production of the therapeutic
genes in the affected target tissues; (ii) to validate the efficacy and
safety of this next-generation gene therapy platform in a preclinical
animal model for Pompe disease. This HERCULES proposal is highly
focused and may ultimately pave the way towards a phase I clinical
trial in patients suffering from Pompe disease, which highlights its
translational potential and medical relevance with possibly broad
implications for patients suffering from other rare muscle disorders.
Date:1 Jan 2023 → Today
Keywords:gene therapy
Disciplines:Genetics, Transcription and translation