Zobrazeno 1 - 10
of 35
pro vyhledávání: '"Mercedes Barzi"'
Autor:
Mercedes Barzi, Tong Chen, Trevor J. Gonzalez, Francis P. Pankowicz, Seh Hoon Oh, Helen L. Streff, Alan Rosales, Yunhan Ma, Sabrina Collias, Sarah E. Woodfield, Anna Mae Diehl, Sanjeev A. Vasudevan, Thao N. Galvan, John Goss, Charles A. Gersbach, Beatrice Bissig-Choisat, Aravind Asokan, Karl-Dimiter Bissig
Publikováno v:
Nature Communications, Vol 15, Iss 1, Pp 1-10 (2024)
Abstract Clinical translation of AAV-mediated gene therapy requires preclinical development across different experimental models, often confounded by variable transduction efficiency. Here, we describe a human liver chimeric transgene-free Il2rg −/
Externí odkaz:
https://doaj.org/article/3464bdb424c442108002dbd891da2d8d
Autor:
Ilayda Ates, Callie Stuart, Tanner Rathbone, Mercedes Barzi, Gordon He, Angela M. Major, Vijay Shankar, Rachel A. Lyman, Sidney S. Angner, Trudy F.C. Mackay, Shanthi Srinivasan, Alton Brad Farris, Karl-Dimiter Bissig, Renee N. Cottle
Publikováno v:
Hepatology Communications, Vol 8, Iss 5 (2024)
Background:. We previously demonstrated the successful use of in vivo CRISPR gene editing to delete 4-hydroxyphenylpyruvate dioxygenase (HPD) to rescue mice deficient in fumarylacetoacetate hydrolase (FAH), a disorder known as hereditary tyrosinemia
Externí odkaz:
https://doaj.org/article/bfe468ab97a549e5a155fe6c7c05eae7
Autor:
Marco De Giorgi, Ang Li, Ayrea Hurley, Mercedes Barzi, Alexandria M. Doerfler, Nikitha A. Cherayil, Harrison E. Smith, Jonathan D. Brown, Charles Y. Lin, Karl-Dimiter Bissig, Gang Bao, William R. Lagor
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 21, Iss , Pp 656-669 (2021)
Clinical application of somatic genome editing requires therapeutics that are generalizable to a broad range of patients. Targeted insertion of promoterless transgenes can ensure that edits are permanent and broadly applicable while minimizing risks
Externí odkaz:
https://doaj.org/article/40cb0ffc6dfe4a53a49a6c7a56786698
Autor:
Beatrice Bissig-Choisat, Michele Alves-Bezerra, Barry Zorman, Scott A. Ochsner, Mercedes Barzi, Xavier Legras, Diane Yang, Malgorzata Borowiak, Adam M. Dean, Robert B. York, N. Thao N. Galvan, John Goss, William R. Lagor, David D. Moore, David E. Cohen, Neil J. McKenna, Pavel Sumazin, Karl-Dimiter Bissig
Publikováno v:
JHEP Reports, Vol 3, Iss 3, Pp 100281- (2021)
Background & Aims: The accumulation of neutral lipids within hepatocytes underlies non-alcoholic fatty liver disease (NAFLD), which affects a quarter of the world’s population and is associated with hepatitis, cirrhosis, and hepatocellular carcinom
Externí odkaz:
https://doaj.org/article/7ba96564fd1546cca46ae8ba9c189a39
Autor:
Robert L. Kruse, Mercedes Barzi, Xavier Legras, Francis P. Pankowicz, Nika Furey, Lan Liao, Janming Xu, Beatrice Bissig-Choisat, Betty L. Slagle, Karl-Dimiter Bissig
Publikováno v:
JHEP Reports, Vol 3, Iss 2, Pp 100252- (2021)
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 model containing an integrated overlength 1.3-mer construct has yielde
Externí odkaz:
https://doaj.org/article/3ed29fbe45da41f499709b7abe8d5061
Autor:
Robert L. Kruse, Thomas Shum, Xavier Legras, Mercedes Barzi, Frank P. Pankowicz, Stephen Gottschalk, Karl-Dimiter Bissig
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 7, Iss C, Pp 32-41 (2017)
Current therapies against hepatitis B virus (HBV) do not reliably cure chronic infection, necessitating new therapeutic approaches. The T cell response can clear HBV during acute infection, and the adoptive transfer of antiviral T cells during bone m
Externí odkaz:
https://doaj.org/article/0eda57cb833d4cb99a64fad628fb7cef
Autor:
Mercedes Barzi, Francis P. Pankowicz, Barry Zorman, Xing Liu, Xavier Legras, Diane Yang, Malgorzata Borowiak, Beatrice Bissig-Choisat, Pavel Sumazin, Feng Li, Karl-Dimiter Bissig
Publikováno v:
Nature Communications, Vol 8, Iss 1, Pp 1-9 (2017)
Human liver chimeric mice are increasingly used for drug testing in preclinical development, but express residual murine p450 cytochromes. Here the authors generate mice lacking the Por gene in the liver, and show that human cytochrome metabolism is
Externí odkaz:
https://doaj.org/article/3e218892915b40fa9da11819b0704336
Autor:
Francis P. Pankowicz, Mercedes Barzi, Xavier Legras, Leroy Hubert, Tian Mi, Julie A. Tomolonis, Milan Ravishankar, Qin Sun, Diane Yang, Malgorzata Borowiak, Pavel Sumazin, Sarah H. Elsea, Beatrice Bissig-Choisat, Karl-Dimiter Bissig
Publikováno v:
Nature Communications, Vol 7, Iss 1, Pp 1-6 (2016)
Hereditary tyrosinaemia type I is caused by a gene defect that leads to a lethal accumulation of toxic metabolites in the liver. Here the authors use CRISPR/Cas9 to 'cure' the disease in mice by inactivating another gene, rather than targeting the di
Externí odkaz:
https://doaj.org/article/c9a870f56fb24cb1bba81c0bca196a4d
Autor:
Mercedes Barzi, Francis P. Pankowicz, Barry Zorman, Xing Liu, Xavier Legras, Diane Yang, Malgorzata Borowiak, Beatrice Bissig-Choisat, Pavel Sumazin, Feng Li, Karl-Dimiter Bissig
Publikováno v:
Nature Communications, Vol 8, Iss 1, Pp 1-1 (2017)
An incorrect version of the Supplementary Information was inadvertently published with this Article where the wrong file was included. The HTML has been updated to include the correct version of the Supplementary Information.
Externí odkaz:
https://doaj.org/article/e4c3baa00a464fb7be24791025631cf9
Autor:
Mercedes Barzi, Collin G. Johnson, Tong Chen, Ramona M. Rodriguiz, Madeline Hemmingsen, Trevor J. Gonzalez, Alan Rosales, James Beasley, Cheryl K. Peck, Yunhan Ma, Ashlee R. Stiles, Timothy C. Wood, Raquel Maeso-Diaz, Anna Mae Diehl, Sarah P. Young, Jeffrey I. Everitt, William C. Wetsel, William R. Lagor, Beatrice Bissig-Choisat, Aravind Asokan, Areeg El-Gharbawy, Karl-Dimiter Bissig
Publikováno v:
Science Translational Medicine. 15
Glutaric aciduria type I (GA-1) is an inborn error of metabolism with a severe neurological phenotype caused by the deficiency of glutaryl–coenzyme A dehydrogenase (GCDH), the last enzyme of lysine catabolism. Current literature suggests that toxic