[HTML][HTML] Reprogramming metabolic pathways in vivo with CRISPR/Cas9 genome editing to treat hereditary tyrosinaemia
Many metabolic liver disorders are refractory to drug therapy and require orthotopic liver
transplantation. Here we demonstrate a new strategy, which we call metabolic pathway …
transplantation. Here we demonstrate a new strategy, which we call metabolic pathway …
[HTML][HTML] Development and rescue of human familial hypercholesterolaemia in a xenograft mouse model
…, PK Saha, L Chen, P Bell, FP Pankowicz… - Nature …, 2015 - nature.com
Diseases of lipid metabolism are a major cause of human morbidity, but no animal model
entirely recapitulates human lipoprotein metabolism. Here we develop a xenograft mouse …
entirely recapitulates human lipoprotein metabolism. Here we develop a xenograft mouse …
P450-humanized and human liver chimeric mouse models for studying xenobiotic metabolism and toxicity
…, N Kovalchuk, L Ding, X Fan, FP Pankowicz… - Drug Metabolism and …, 2018 - ASPET
Preclinical evaluation of drug candidates in experimental animal models is an essential step
in drug development. Humanized mouse models have emerged as a promising alternative …
in drug development. Humanized mouse models have emerged as a promising alternative …
[HTML][HTML] A novel humanized mouse lacking murine P450 oxidoreductase for studying human drug metabolism
Only one out of 10 drugs in development passes clinical trials. Many fail because experimental
animal models poorly predict human xenobiotic metabolism. Human liver chimeric mice …
animal models poorly predict human xenobiotic metabolism. Human liver chimeric mice …
CRISPR/Cas9: at the cutting edge of hepatology
FP Pankowicz, KE Jarrett, WR Lagor, KD Bissig - Gut, 2017 - gut.bmj.com
Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/Cas9 genome
engineering has revolutionised biomedical science and we are standing on the cusp of medical …
engineering has revolutionised biomedical science and we are standing on the cusp of medical …
[HTML][HTML] A humanized mouse model for adeno-associated viral gene therapy
Clinical translation of AAV-mediated gene therapy requires preclinical development across
different experimental models, often confounded by variable transduction efficiency. Here, …
different experimental models, often confounded by variable transduction efficiency. Here, …
Rapid disruption of genes specifically in livers of mice using multiplex CRISPR/Cas9 editing
Background & aims Despite advances in gene editing technologies, generation of tissue-specific
knockout mice is time-consuming. We used CRISPR/Cas9-mediated genome editing to …
knockout mice is time-consuming. We used CRISPR/Cas9-mediated genome editing to …
[HTML][HTML] A hepatitis B virus transgenic mouse model with a conditional, recombinant, episomal genome
Background & Aims Development of new and more effective therapies against hepatitis B
virus (HBV) is limited by the lack of suitable small animal models. The HBV transgenic mouse …
virus (HBV) is limited by the lack of suitable small animal models. The HBV transgenic mouse …
[CITATION][C] Long-Term Consequences of CRISPR/Cas9 Gene Editing in a Mouse Model for Hereditary Tyrosinemia Type I
FP Pankowicz, M Barzi… - MOLECULAR …, 2019 - CELL PRESS 50 HAMPSHIRE ST …
[CITATION][C] CRISPR/Cas9-MEDIATED CASPASE 2 DELETION IN HEPATOCYTES MODULATES CHOLESTEROL METABOLISM AND ALLEVIATES INSULIN …
GD Dalton, M Jewell, FP Pankowicz… - …, 2019 - … ST, HOBOKEN 07030-5774, NJ USA