Results 101 to 110 of about 1,520,545 (236)

In vivo precision base editing to rescue mouse models of disease

open access: yesMolecular Therapy: Nucleic Acids
CRISPR base editing enables precise, irreversible base conversions without inducing double-stranded breaks (DSBs) and has gained significant attention in recent years. By converting cytosine to thymine (C→T) or adenine to guanine (A→G), base editors (BEs)
Aaron Schindeler   +5 more
doaj   +1 more source

Choosing a pediatric recipient for orthotopic liver transplantation [PDF]

open access: yes, 1987
Between March 3, 1981, and June 1, 1984, 216 children were evaluated for orthotopic liver transplantation. Of the 216 patients, 117 (55%) had recelved at least one liver transplant by June 1, 1985. Fifty-five (25%) died before transplantation.
Andrew H. Urbach   +31 more
core   +1 more source

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

open access: yesCells
Hepatocyte transplantation (HTx) combined with ex vivo gene therapy has garnered significant interest due to its potential for treating many inherited metabolic liver diseases.
Justin Gibson   +6 more
doaj   +1 more source

Hormonally and chemically defined expansion conditions for organoids of biliary tree Stem Cells

open access: yesBioactive Materials
Wholly defined ex vivo expansion conditions for biliary tree stem cell (BTSC) organoids were established, consisting of a defined proliferative medium (DPM) used in combination with soft hyaluronan hydrogels.
Wencheng Zhang   +13 more
doaj   +1 more source

Evaluation of Neonatal Screening Programs for Tyrosinemia Type 1 Worldwide

open access: yesInternational Journal of Neonatal Screening
In The Netherlands, newborn screening (NBS) for tyrosinemia type 1 (TT1) uses dried blood spot (DBS) succinylacetone (SUAC) as a biomarker. However, high false-positive (FP) rates and a false-negative (FN) case show that the Dutch TT1 NBS protocol is ...
Allysa M. Kuypers   +9 more
doaj   +1 more source

Evolution of Liver Transplantation [PDF]

open access: yes, 1982
Abouna   +142 more
core   +1 more source

Orthotopic liver transplantation in children. Two-year experience with 47 patients [PDF]

open access: yes, 1984
During a 24-month period (May 1981 to May 1983), 47 pediatric patients (ranging in age from 7 months to 18 years) underwent orthotopic liver transplantation using cyclosporine and prednisone.
Gartner, JC   +5 more
core  

HEREDITARY TYROSINEMIA TYPE I

open access: yesJournal of Pediatric Gastroenterology and Nutrition, 1991
F. J. van Spronsen   +4 more
openaire   +2 more sources

A Rapid Screening Test on Dried Blood for the Neonatal Diagnosis of Tyrosinemia Type I. [PDF]

open access: yesIran J Pediatr, 2016
Bodaghkhan F   +7 more
europepmc   +1 more source

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