VanLith C, Guthman R, Nicolas CT, Allen K, Du Z, Joo DJ, et al. Curative ex vivo hepatocyte-directed gene editing in a mouse model of hereditary Tyrosinemia Type 1. Hum Gene Ther. 2018;29:1315–26.
Article CAS PubMed PubMed Central Google Scholar
Mingozzi F, High KA. Immune responses to AAV in clinical trials. Curr Gene Ther. 2011;11:321–30.
Article CAS PubMed Google Scholar
Mingozzi F, High KA. Immune responses to AAV vectors: Overcoming barriers to successful gene therapy. Blood. 2013;122:23–36.
Article CAS PubMed PubMed Central Google Scholar
Tran ND, Porada CD, Almeida-Porada G, Glimp HA, Anderson WF, Zanjani ED. Induction of stable prenatal tolerance to beta-galactosidase by in utero gene transfer into preimmune sheep fetuses. Blood. 2001;97:3417–23.
Article CAS PubMed Google Scholar
Porada CD, Park PJ, Almeida-Porada G, Liu W, Ozturk F, Glimp HA, et al. Gestational age of recipient determines pattern and level of transgene expression following in utero retroviral gene transfer. Mol Ther. 2005;11:284–93.
Article CAS PubMed Google Scholar
Flake AW. Genetic therapies for the fetus. Clin Obstet Gynecol. 2002;45:684–96.
Alapati D, Zacharias WJ, Hartman HA, Rossidis AC, Stratigis JD, Ahn NJ, et al. In utero gene editing for monogenic lung disease. Sci Transl Med. 2019;11:eaav8375.
Article PubMed PubMed Central Google Scholar
Rossidis AC, Stratigis JD, Chadwick AC, Hartman HA, Ahn NJ, Li H, et al. In utero CRISPR-mediated therapeutic editing of metabolic genes. Nat Med. 2018;24:1513–8.
Article CAS PubMed PubMed Central Google Scholar
Lindblad B, Lindstedt S, Steen G. On the enzymic defects in hereditary tyrosinemia. Proc Natl Acad Sci USA. 1977;74:4641–5.
Article CAS PubMed PubMed Central Google Scholar
Endo F, Sun MS. Tyrosinaemia type I and apoptosis of hepatocytes and renal tubular cells. J Inherit Metab Dis. 2002;25:227–34.
Article CAS PubMed Google Scholar
Grompe M. The pathophysiology and treatment of hereditary tyrosinemia type 1. Semin Liver Dis. 2001;21:563–71.
Article CAS PubMed Google Scholar
Gilgenkrantz H. Rodent models of liver repopulation. Methods Mol Biol. 2010;640:475–90.
Mao Z, Bozzella M, Seluanov A, Gorbunova V. DNA repair by nonhomologous end joining and homologous recombination during cell cycle in human cells. Cell Cycle. 2008;7:2902–6.
Article CAS PubMed Google Scholar
VanLith CJ, Guthman RM, Nicolas CT, Allen KL, Liu Y, Chilton JA, et al. Ex vivo hepatocyte reprograming promotes homology-directed DNA repair to correct metabolic disease in mice after transplantation. Hepatol Commun. 2019;3:558–73.
Article CAS PubMed PubMed Central Google Scholar
Yin H, Song CQ, Dorkin JR, Zhu LJ, Li Y, Wu Q, et al. Therapeutic genome editing by combined viral and non-viral delivery of CRISPR system components in vivo. Nat Biotechnol. 2016;34:328–33.
Article CAS PubMed PubMed Central Google Scholar
Kreutz C, MacNelly S, Follo M, Waldin A, Binninger-Lacour P, Timmer J, et al. Hepatocyte ploidy is a diversity factor for liver homeostasis. Front Physiol. 2017;8:862.
Article PubMed PubMed Central Google Scholar
Chen Y, Hata T, Rehman F, Kang L, Yang L, Kim BYS, et al. Visualization of hepatocellular regeneration in mice after partial hepatectomy. J Surg Res. 2019;235:494–500.
Tao Y, Wang M, Chen E, Tang H. Liver regeneration: Analysis of the main relevant signaling molecules. Mediators Inflamm. 2017;2017:4256352.
Article PubMed PubMed Central Google Scholar
Iyama T, Wilson DM 3rd. DNA repair mechanisms in dividing and non-dividing cells. DNA Repair (Amst). 2013;12:620–36.
Michalopoulos GK. Liver regeneration. J Cellular Physiol. 2007;213:286–300.
Cerone R, Holme E, Schiaffino MC, Caruso U, Maritano L, Romano C. Tyrosinemia type III: Diagnosis and ten-year follow-up. Acta Paediatr. 1997;86:1013–5.
Article CAS PubMed Google Scholar
Ruetschi U, Cerone R, Perez-Cerda C, Schiaffino MC, Standing S, Ugarte M, et al. Mutations in the 4-hydroxyphenylpyruvate dioxygenase gene (HPD) in patients with tyrosinemia type III. Hum Genet. 2000;106:654–62.
Article CAS PubMed Google Scholar
Chinsky JM, Singh R, Ficicioglu C, van Karnebeek CDM, Grompe M, Mitchell G, et al. Diagnosis and treatment of tyrosinemia type I: A US and Canadian consensus group review and recommendations. Genet Med. 2017;19:1380–95.
Vermeulen C, Geeven G, de Wit E, Verstegen M, Jansen RPM, van Kranenburg M, et al. Sensitive monogenic noninvasive prenatal diagnosis by targeted haplotyping. Am J Hum Genet. 2017;101:326–39.
Article CAS PubMed PubMed Central Google Scholar
Rafati M, Mohamadhashem F, Hoseini A, Ramandi SD, Ghaffari SR. Prenatal diagnosis of tyrosinemia type 1 using next generation sequencing. Fetal Pediatr Pathol. 2016;35:282–5.
Article CAS PubMed Google Scholar
David AL, Peebles D. Gene therapy for the fetus: Is there a future? Best Pract Res Clin Obstet Gynaecol. 2008;22:203–18.
Kim CH. Homeostatic and pathogenic extramedullary hematopoiesis. J Blood Med. 2010;1:13–9.
Article PubMed PubMed Central Google Scholar
Dutta S, Sengupta P. Men and mice: Relating their ages. Life Sci. 2016;152:244–8.
Article CAS PubMed Google Scholar
Krishnan A, Samtani R, Dhanantwari P, Lee E, Yamada S, Shiota K, et al. A detailed comparison of mouse and human cardiac development. Pediatr Res. 2014;76:500–7.
Article PubMed PubMed Central Google Scholar
Pressler R, Auvin S. Comparison of brain maturation among species: An example in translational research suggesting the possible use of bumetanide in newborn. Front Neurol. 2013;4:36.
Article PubMed PubMed Central Google Scholar
Li N, Gou S, Wang J, Zhang Q, Huang X, Xie J, et al. CRISPR/Cas9-mediated gene correction in newborn rabbits with hereditary tyrosinemia type I. Mol Ther. 2020;29:1001–15.
Article PubMed PubMed Central Google Scholar
Lin S, Staahl BT, Alla RK, Doudna JA. Enhanced homology-directed human genome engineering by controlled timing of CRISPR/Cas9 delivery. Elife. 2014;3:e04766.
Article PubMed PubMed Central Google Scholar
Jackman J, O’Connor PM. Methods for synchronizing cells at specific stages of the cell cycle. Curr Protoc Cell Biol. 2001;Chapter 8:Unit 8.3.
Thyagarajan B, Cruise JL, Campbell C. Elevated levels of homologous DNA recombination activity in the regenerating rat liver. Somat Cell Mol Genet. 1996;22:31–9.
Article CAS PubMed Google Scholar
Xiong W, MacColl Garfinkel AE, Li Y, Benowitz LI, Cepko CL. NRF2 promotes neuronal survival in neurodegeneration and acute nerve damage. J Clin Invest. 2015;125:1433–45.
Article PubMed PubMed Central Google Scholar
Ran FA, Cong L, Yan WX, Scott DA, Gootenberg JS, Kriz AJ, et al. In vivo genome editing using Staphylococcus aureus Cas9. Nature. 2015;520:186–91.
Article CAS PubMed PubMed Central Google Scholar
Grompe M, al-Dhalimy M, Finegold M, Ou CN, Burlingame T, Kennaway NG, et al. Loss of fumarylacetoacetate hydrolase is responsible for the neonatal hepatic dysfunction phenotype of lethal albino mice. Genes Dev. 1993;7:2298–307.
Article CAS PubMed Google Scholar
Nijagal A, Le T, Wegorzewska M, Mackenzie TC. A mouse model of in utero transplantation. J Vis Exp. 2011;47:2303.
Waddington SN, Mitrophanous KA, Ellard FM, Buckley SM, Nivsarkar M, Lawrence L, et al. Long-term transgene expression by administration of a lentivirus-based vector to the fetal circulation of immuno-competent mice. Gene Ther. 2003;10:1234–40.
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