Article

Targeted delivery of acid alpha-glucosidase corrects skeletal muscle phenotypes in Pompe disease mice

2020-04-23

Abstract excerpt

Lysosomal diseases are a class of genetic disorders predominantly caused by loss of lysosomal hydrolases, leading to lysosomal and cellular dysfunction. Enzyme Replacement Therapy (ERT), where recombinant enzyme is given intravenously, internalized by cells, and trafficked to the lysosome, has been applied to treat several lysosomal diseases. However, current ERT regimens do not correct disease phenotypes in all a...

Topics

Open a Topic to create a Post that cites this publication.

Identifiers and source

Literature Corpus work: 46a45a3a-adcd-597f-b48a-c4240b7af315
DOI: 10.1101/2020.04.22.051672

Open publication

Related research

Semantic proximity does not establish scientific evidence.

Click a neighbor to travelStep 1 · 12 closest

Targeted delivery of acid alpha-glucosidase corrects skeletal muscle phenotypes in Pompe disease miceDOI 10.1101/2020.04.22.051672Read

Advancements in AAV-mediated Gene Therapy for Pompe DiseaseSemantic proximity Read

Gene Therapy for Pompe Disease: The Time is nowSemantic proximity Read

Screening chimeric GAA variants in preclinical study results in hematopoietic stem cell gene therapy candidate vectors for Pompe diseaseSemantic proximity Read

Hepatic expression of GAA results in enhanced enzyme bioavailability in mice and non-human primatesSemantic proximity Read

Pompe Disease: A Review of Diagnosis, Molecular Genetics, and Treatment Management.Semantic proximity Read

Screening of Chimeric GAA Variants in a Preclinical Study of Pompe Disease Results in Candidate Vector for Hematopoietic Stem Cell Gene TherapySemantic proximity Read

What’s new and what’s next for gene therapy in Pompe disease?Semantic proximity Read

Gene Therapy Developments for Pompe DiseaseSemantic proximity Read

Muscle-specific, liver-detargeted adeno-associated virus gene therapy rescues Pompe phenotype in adult and neonate Gaa-/- mice.Semantic proximity Read

Preclinical lentiviral vector-mediated hematopoietic stem and progenitor cell gene therapy corrects Pompe disease-related muscle and neurological manifestationsSemantic proximity Read

Conditional tissue-specific expression of the acid alpha-glucosidase (GAA) gene in the GAA knockout mice: implications for therapySemantic proximity Read

Rescue of Advanced Pompe Disease in Mice with Hepatic Expression of Secretable Acid α-Glucosidase.Semantic proximity Read