Results 141 to 150 of about 3,137 (203)

Validation of Clinical-Grade Electroporation Systems for CRISPR-Cas9-Mediated Gene Therapy in Primary Hepatocytes for the Correction of Inherited Metabolic Liver Disease. [PDF]

Cells
Gibson J, Dhungana A, Pokhrel M, Arthur B, Suresh P, Adebayo O, Cottle RN.   +6 more
europepmc   +1 more source

CRISPR/Cas9 gene therapy increases the risk of tumorigenesis in the mouse model of hereditary tyrosinemia type I. [PDF]

JHEP Rep
Chen T, Barzi M, Furey N, Kim HR, Pankowicz FP, Legras X, Elsea SH, Hurley AE, Yang D, Wheeler DA, Borowiak M, Bissig-Choisat B, Sumazin P, Bissig KD.   +13 more
europepmc   +1 more source

Inborn Errors of Amino Acid Metabolism Revisited: Clinical Implications and Insights into Current Therapies. [PDF]

J Clin Med
Shakerdi AL, Nerney D, Molloy EJ, Knerr I.   +3 more
europepmc   +1 more source

Mouse Models for Chronic Hepatitis B: Old Challenges, Novel Approaches. [PDF]

Cell Mol Gastroenterol Hepatol
Broeckhoven E, Dallmeier K.
europepmc   +1 more source

Estrogen-Related Receptor Potential Target Genes in Silkworm (<i>Bombyx mori</i>): Insights into Metabolic Regulation. [PDF]

Insects
Hou L, Wu J, Liu D, Xu H, Yao H, Liang Y, Xia Q, Lin P, Shen G.   +8 more
europepmc   +1 more source

Kinetic studies of the hydrolysis of 3,5-diketo acids by fumarylacetoacetate fumarylhydrolase. [PDF]

, 1976
Angus, Ronald H.
core   +1 more source

Efficient AAV8 delivery to the liver via isolated hepatic perfusion and analysis of hepatic lobule transduction patterns. [PDF]

Liver Res
Wang C, Fan M, Liu J, Guo X, Tan Z, Huang X, Li Z, Liu X, Zhang Y, Feng J, Fang R, Wang L, Ke Q.   +12 more
europepmc   +1 more source

Slow-onset inhibition of fumarylacetoacetate hydrolase by phosphinate mimics of the tetrahedral intermediate: kinetics, crystal structure and pharmacokinetics. [PDF]

Biochem J, 2007
Bateman RL, Ashworth J, Witte JF, Baker LJ, Bhanumoorthy P, Timm DE, Hurley TD, Grompe M, McClard RW.   +8 more
europepmc   +1 more source

Metabolic characteristics in hepatocellular carcinoma: amino acid metabolic reprogramming. [PDF]

Mol Cancer
Zhou R, Li Y, Li G, Li Y, Luo L, Wang B, Wang L.   +6 more
europepmc   +1 more source

In Vivo Confocal Microscopy and Anterior Segment Optical Coherence Tomography Features of Corneal Pseudodendritic Lesions in Hereditary Tyrosinemia Type 1. [PDF]

Am J Case Rep
Bruno R, Donatelli S, Mastropasqua L, Nubile M.   +3 more
europepmc   +1 more source