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View article: Ex vivo precision-cut liver slices model disease phenotype and monitor therapeutic response for liver monogenic diseases
Ex vivo precision-cut liver slices model disease phenotype and monitor therapeutic response for liver monogenic diseases Open
Background In academic research and the pharmaceutical industry, in vitro cell lines and in vivo animal models are considered as gold standards in modelling diseases and assessing therapeutic efficacy. However, both models have intrinsic l…
View article: mRNA therapy corrects defective glutathione metabolism and restores ureagenesis in preclinical argininosuccinic aciduria
mRNA therapy corrects defective glutathione metabolism and restores ureagenesis in preclinical argininosuccinic aciduria Open
The urea cycle enzyme argininosuccinate lyase (ASL) enables the clearance of neurotoxic ammonia and the biosynthesis of arginine. Patients with ASL deficiency present with argininosuccinic aciduria, an inherited metabolic disease with hype…
View article: Ex vivo precision-cut liver slices model disease phenotype and monitor therapeutic response for liver monogenic diseases
Ex vivo precision-cut liver slices model disease phenotype and monitor therapeutic response for liver monogenic diseases Open
Background In academic research and the pharmaceutical industry, in vitro cell lines and in vivo animal models are considered as gold standards in modelling diseases and assessing therapeutic efficacy. However, both models have intrinsic …
View article: The incidence of movement disorder increases with age and contrasts with subtle and limited neuroimaging abnormalities in argininosuccinic aciduria
The incidence of movement disorder increases with age and contrasts with subtle and limited neuroimaging abnormalities in argininosuccinic aciduria Open
Argininosuccinate lyase (ASL) is integral to the urea cycle detoxifying neurotoxic ammonia and the nitric oxide (NO) biosynthesis cycle. Inherited ASL deficiency causes argininosuccinic aciduria (ASA), a rare disease with hyperammonemia an…
View article: Induction of long-term tolerance to a specific antigen using anti-CD3 lipid nanoparticles following gene therapy
Induction of long-term tolerance to a specific antigen using anti-CD3 lipid nanoparticles following gene therapy Open
Development of factor VIII (FVIII) inhibitors is a serious complication in the treatment of hemophilia A (HemA) patients. In clinical trials, anti-CD3 antibody therapy effectively modulates the immune response of allograft rejection or aut…
View article: Macrophage inhibitor clodronate enhances liver transduction of lentiviral but not AAV vectors or mRNA lipid nanoparticles<i>in vivo</i>
Macrophage inhibitor clodronate enhances liver transduction of lentiviral but not AAV vectors or mRNA lipid nanoparticles<i>in vivo</i> Open
Recently approved adeno-associated viral (AAV) vectors for liver monogenic diseases hemophilia A and B are exemplifying the success of liver-directed viral gene therapy. In parallel, additional strategies are rapidly emerging to overcome s…
View article: <i>Ex vivo</i>primary liver sections recapitulate disease phenotype and therapeutic rescue for liver monogenic diseases
<i>Ex vivo</i>primary liver sections recapitulate disease phenotype and therapeutic rescue for liver monogenic diseases Open
In academic research and the pharmaceutical industry, in vitro single cell line cultures and in vivo animal models are considered as gold standards in modelling diseases and assessing therapeutic efficacy. However, both models have limitat…
View article: mRNA therapy restores ureagenesis and corrects glutathione metabolism in argininosuccinic aciduria
mRNA therapy restores ureagenesis and corrects glutathione metabolism in argininosuccinic aciduria Open
Argininosuccinate lyase (ASL) is a key enzyme integral to the hepatic urea cycle which is required for ammonia detoxification, and the citrulline-nitric oxide (NO) cycle for NO production. ASL deficient patients present with argininosuccin…
View article: Systemic modified messenger RNA for replacement therapy in alpha 1-antitrypsin deficiency
Systemic modified messenger RNA for replacement therapy in alpha 1-antitrypsin deficiency Open
View article: Treatment of Hemophilia A Using Factor VIII Messenger RNA Lipid Nanoparticles
Treatment of Hemophilia A Using Factor VIII Messenger RNA Lipid Nanoparticles Open