المؤلفون: 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

المصدر: Hepatology Communications, Vol 8, Iss 5 (2024)

مصطلحات موضوعية: Diseases of the digestive system. Gastroenterology, RC799-869

الوصف: 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 type 1 (HT1). The aim of this study was to develop an ex vivo gene-editing protocol and apply it as a cell therapy for HT1. Methods:. We isolated hepatocytes from wild-type (C57BL/6J) and Fah −/− mice and then used an optimized electroporation protocol to deliver Hpd-targeting CRISPR-Cas9 ribonucleoproteins into hepatocytes. Next, hepatocytes were transiently incubated in cytokine recovery media formulated to block apoptosis, followed by splenic injection into recipient Fah −/− mice. Results:. We observed robust engraftment and expansion of transplanted gene-edited hepatocytes from wild-type donors in the livers of recipient mice when transient incubation with our cytokine recovery media was used after electroporation and negligible engraftment without the media (mean: 46.8% and 0.83%, respectively; p=0.0025). Thus, the cytokine recovery medium was critical to our electroporation protocol. When hepatocytes from Fah −/− mice were used as donors for transplantation, we observed 35% and 28% engraftment for Hpd-Cas9 ribonucleoproteins and Cas9 mRNA, respectively. Tyrosine, phenylalanine, and biochemical markers of liver injury normalized in both Hpd-targeting Cas9 ribonucleoprotein and mRNA groups independent of induced inhibition of Hpd through nitisinone, indicating correction of disease indicators in Fah −/− mice. Conclusions:. The successful liver cell therapy for HT1 validates our protocol and, despite the known growth advantage of HT1, showcases ex vivo gene editing using electroporation in combination with liver cell therapy to cure a disease model. These advancements underscore the potential impacts of electroporation combined with transplantation as a cell therapy.

وصف الملف: electronic resource

العلاقة: http://journals.lww.com/10.1097/HC9.0000000000000424Test; https://doaj.org/toc/2471-254XTest

الوصول الحر: https://doaj.org/article/bfe468ab97a549e5a155fe6c7c05eae7Test

المؤلفون: Marjan Afrouzian, Nicolas Kozakowski, Helen Liapis, Verena Broecker, Luan Truong, Carmen Avila-Casado, Heinz Regele, Surya Seshan, Josephine M. Ambruzs, Alton Brad Farris, David Buob, Praveen N. Chander, Lukman Cheraghvandi, Marian C. Clahsen-van Groningen, Stanley de Almeida Araujo, Dilek Ertoy Baydar, Mark Formby, Danica Galesic Ljubanovic, Loren Herrera Hernandez, Eva Honsova, Nasreen Mohamed, Yasemin Ozluk, Marion Rabant, Virginie Royal, Heather L. Stevenson, Maria Fernanda Toniolo, Diana Taheri

المصدر: Transplant International, Vol 36 (2023)

مصطلحات موضوعية: Delphi, Banff, thrombotic microangiopathy, kidney, transplantation, Specialties of internal medicine, RC581-951

وصف الملف: electronic resource

العلاقة: https://www.frontierspartnerships.org/articles/10.3389/ti.2023.12046/fullTest; https://doaj.org/toc/1432-2277Test

الوصول الحر: https://doaj.org/article/345d0873d1ff40aba7255d74e0f29d74Test

المؤلفون: Marjan Afrouzian, Nicolas Kozakowski, Helen Liapis, Verena Broecker, Luon Truong, Carmen Avila-Casado, Heinz Regele, Surya Seshan, Josephine M. Ambruzs, Alton Brad Farris, David Buob, Praveen N. Chander, Lukman Cheraghvandi, Marian C. Clahsen-van Groningen, Stanley de Almeida Araujo, Dilek Ertoy Baydar, Mark Formby, Danica Galesic Ljubanovic, Loren Herrera Hernandez, Eva Honsova, Nasreen Mohamed, Yasemin Ozluk, Marion Rabant, Virginie Royal, Heather L. Stevenson, Maria Fernanda Toniolo, Diana Taheri

المصدر: Transplant International, Vol 36 (2023)

مصطلحات موضوعية: thrombotic microangiopathy, kidney, transplant, pathology criteria, Delphi, Banff, Specialties of internal medicine, RC581-951

وصف الملف: electronic resource

العلاقة: https://www.frontierspartnerships.org/articles/10.3389/ti.2023.12047/fullTest; https://doaj.org/toc/1432-2277Test

الوصول الحر: https://doaj.org/article/c7614e166fa340a1843e277bf33b05e0Test

المؤلفون: Marjan Afrouzian, Nicolas Kozakowski, Helen Liapis, Verena Broecker, Luan Truong, Carmen Avila-Casado, Heinz Regele, Surya Seshan, Josephine M. Ambruzs, Alton Brad Farris, David Buob, Praveen N. Chander, Lukman Cheraghvandi, Marian C. Clahsen-van Groningen, Stanley de Almeida Araujo, Dilek Ertoy Baydar, Mark Formby, Danica Galesic Ljubanovic, Loren Herrera Hernandez, Eva Honsova, Nasreen Mohamed, Yasemin Ozluk, Marion Rabant, Virginie Royal, Heather L. Stevenson, Maria Fernanda Toniolo, Diana Taheri

المصدر: Transplant International, Vol 36 (2023)

مصطلحات موضوعية: Delphi, Banff, thrombotic microangiopathy, kidney, transplantation, Specialties of internal medicine, RC581-951

الوصف: The Thrombotic Microangiopathy Banff Working Group (TMA-BWG) was formed in 2015 to survey current practices and develop minimum diagnostic criteria (MDC) for renal transplant TMA (Tx-TMA). To generate consensus among pathologists and nephrologists, the TMA BWG designed a 3-Phase study. Phase I of the study is presented here. Using the Delphi methodology, 23 panelists with >3 years of diagnostic experience with Tx-TMA pathology listed their MDC suggesting light, immunofluorescence, and electron microscopy lesions, clinical and laboratory information, and differential diagnoses. Nine rounds (R) of consensus resulted in MDC validated during two Rs using online evaluation of whole slide digital images of 37 biopsies (28 TMA, 9 non-TMA). Starting with 338 criteria the process resulted in 24 criteria and 8 differential diagnoses including 18 pathologic, 2 clinical, and 4 laboratory criteria. Results show that 3/4 of the panelists agreed on the diagnosis of 3/4 of cases. The process also allowed definition refinement for 4 light and 4 electron microscopy lesions. For the first time in Banff classification, the Delphi methodology was used to generate consensus. The study shows that Delphi is a democratic and cost-effective method allowing rapid consensus generation among numerous physicians dealing with large number of criteria in transplantation.

وصف الملف: electronic resource

العلاقة: https://www.frontierspartnerships.org/articles/10.3389/ti.2023.11589/fullTest; https://doaj.org/toc/1432-2277Test

الوصول الحر: https://doaj.org/article/7676434f4c7e41579e979276c1818a96Test

المؤلفون: Marjan Afrouzian, Nicolas Kozakowski, Helen Liapis, Verena Broecker, Luon Truong, Carmen Avila-Casado, Heinz Regele, Surya Seshan, Josephine M. Ambruzs, Alton Brad Farris, David Buob, Praveen N. Chander, Lukman Cheraghvandi, Marian C. Clahsen-van Groningen, Stanley de Almeida Araujo, Dilek Ertoy Baydar, Mark Formby, Danica Galesic Ljubanovic, Loren Herrera Hernandez, Eva Honsova, Nasreen Mohamed, Yasemin Ozluk, Marion Rabant, Virginie Royal, Heather L. Stevenson, Maria Fernanda Toniolo, Diana Taheri

المصدر: Transplant International, Vol 36 (2023)

مصطلحات موضوعية: thrombotic microangiopathy, kidney, transplant, pathology criteria, Delphi, Banff, Specialties of internal medicine, RC581-951

الوصف: The Banff community summoned the TMA Banff Working Group to develop minimum diagnostic criteria (MDC) and recommendations for renal transplant TMA (Tx-TMA) diagnosis, which currently lacks standardized criteria. Using the Delphi method for consensus generation, 23 nephropathologists (panelists) with >3 years of diagnostic experience with Tx-TMA were asked to list light, immunofluorescence, and electron microscopic, clinical and laboratory criteria and differential diagnoses for Tx-TMA. Delphi was modified to include 2 validations rounds with histological evaluation of whole slide images of 37 transplant biopsies (28 TMA and 9 non-TMA). Starting with 338 criteria in R1, MDC were narrowed down to 24 in R8 generating 18 pathological, 2 clinical, 4 laboratory criteria, and 8 differential diagnoses. The panelists reached a good level of agreement (70%) on 76% of the validated cases. For the first time in Banff classification, Delphi was used to reach consensus on MDC for Tx-TMA. Phase I of the study (pathology phase) will be used as a model for Phase II (nephrology phase) for consensus regarding clinical and laboratory criteria. Eventually in Phase III (consensus of the consensus groups) and the final MDC for Tx-TMA will be reported to the transplantation community.

وصف الملف: electronic resource

العلاقة: https://www.frontierspartnerships.org/articles/10.3389/ti.2023.11590/fullTest; https://doaj.org/toc/1432-2277Test

الوصول الحر: https://doaj.org/article/e101ede6be324704a1a6619ba7988739Test

المؤلفون: Ravi Chakra Turaga, Ganesh Satyanarayana, Malvika Sharma, Jenny J. Yang, Shiyuan Wang, Chunfeng Liu, Sun Li, Hua Yang, Hans Grossniklaus, Alton Brad Farris, Jordi Gracia-Sancho, Zhi-Ren Liu

المصدر: Communications Biology, Vol 4, Iss 1, Pp 1-10 (2021)

مصطلحات موضوعية: Biology (General), QH301-705.5

الوصف: As activated hepatic stellate cells and liver sinusoidal endothelial cells express high levels of integrin α v β3, Turaga et al. present a strategy for treatment of chronic liver disease using their rationally designed protein (ProAgio) which targets integrin α v β3. They find that ProAgio –mediated apoptosis of these disease-associated cells reverses liver fibrosis and ameliorates intrahepatic vascular resistance in a mouse model

وصف الملف: electronic resource

العلاقة: https://doaj.org/toc/2399-3642Test

الوصول الحر: https://doaj.org/article/ab156e8cd1c24f94ba28fcb876a8cb76Test

المؤلفون: Syed H. Zaidi, Tabitha A. Harrison, Amanda I. Phipps, Robert Steinfelder, Quang M. Trinh, Conghui Qu, Barbara L. Banbury, Peter Georgeson, Catherine S. Grasso, Marios Giannakis, Jeremy B. Adams, Elizabeth Alwers, Efrat L. Amitay, Richard T. Barfield, Sonja I. Berndt, Ivan Borozan, Hermann Brenner, Stefanie Brezina, Daniel D. Buchanan, Yin Cao, Andrew T. Chan, Jenny Chang-Claude, Charles M. Connolly, David A. Drew, Alton Brad Farris, Jane C. Figueiredo, Amy J. French, Charles S. Fuchs, Levi A. Garraway, Steve Gruber, Mark A. Guinter, Stanley R. Hamilton, Sophia Harlid, Lawrence E. Heisler, Akihisa Hidaka, John L. Hopper, Wen-Yi Huang, Jeroen R. Huyghe, Mark A. Jenkins, Paul M. Krzyzanowski, Mathieu Lemire, Yi Lin, Xuemei Luo, Elaine R. Mardis, John D. McPherson, Jessica K. Miller, Victor Moreno, Xinmeng Jasmine Mu, Reiko Nishihara, Nickolas Papadopoulos, Danielle Pasternack, Michael J. Quist, Adilya Rafikova, Emma E. G. Reid, Eve Shinbrot, Brian H. Shirts, Lincoln D. Stein, Cherie D. Teney, Lee Timms, Caroline Y. Um, Bethany Van Guelpen, Megan Van Tassel, Xiaolong Wang, David A. Wheeler, Christina K. Yung, Li Hsu, Shuji Ogino, Andrea Gsur, Polly A. Newcomb, Steven Gallinger, Michael Hoffmeister, Peter T. Campbell, Stephen N. Thibodeau, Wei Sun, Thomas J. Hudson, Ulrike Peters

المصدر: Nature Communications, Vol 11, Iss 1, Pp 1-12 (2020)

مصطلحات موضوعية: Science

الوصف: Large scale sequencing study is of paramount importance to unravel the heterogeneity of colorectal cancer. Here, the authors sequenced 205 cancer genes in more than 2000 tumours and identified additional mutated driver genes, determined that mutational burden and specific mutations in TP53 are associated with survival odds.

وصف الملف: electronic resource

العلاقة: https://doaj.org/toc/2041-1723Test

الوصول الحر: https://doaj.org/article/a09dab8e99d844a6a6258b23214b612cTest

المؤلفون: Mani Salarian, Ravi Chakra Turaga, Shenghui Xue, Maysam Nezafati, Khan Hekmatyar, Jingjuan Qiao, Yinwei Zhang, Shanshan Tan, Oluwatosin Y. Ibhagui, Yan Hai, Jibiao Li, Rao Mukkavilli, Malvika Sharma, Pardeep Mittal, Xiaoyi Min, Shella Keilholz, Liqing Yu, Gengshen Qin, Alton Brad Farris, Zhi-Ren Liu, Jenny J. Yang

المصدر: Nature Communications, Vol 10, Iss 1, Pp 1-14 (2019)

مصطلحات موضوعية: Science

الوصف: Non-invasive early diagnosis of liver fibrosis is important to prevent disease progression and direct treatment strategies. Here the authors developed a collagen-targeting contrast agent for the detection of early stage fibrosis and non-alcoholic steatohepatitis by magnetic resonance and tested it in animal models.

وصف الملف: electronic resource

العلاقة: https://doaj.org/toc/2041-1723Test

الوصول الحر: https://doaj.org/article/7815a12c65474deb997bdf57a3b68b7bTest

المؤلفون: Aashiyana F, Koreishi, Rosalynn M, Nazarian, Adam J, Saenz, Veronica E, Klepeis, Anna G, McDonald, Alton Brad, Farris, Robert B, Colvin, Lyn M, Duncan, Rajni V, Mandal, Jonathan, Kay

المصدر: Archives of Pathology & Laboratory Medicine. 133:1943-1948

مصطلحات موضوعية: Adult, Male, Pneumonia, General Medicine, Middle Aged, Nephrogenic Fibrosing Dermopathy, Pathology and Forensic Medicine, Medical Laboratory Technology, Fatal Outcome, Risk Factors, Cause of Death, Thromboembolism, Humans, Female, Autopsy, Aged

الوصف: Context.—Nephrogenic systemic fibrosis (NSF) is a rare but serious disorder initially described as a purely dermatologic process. Isolated autopsy reports have described multiorgan involvement by this disease. Objective.—To further illustrate the varied and systemic involvement of NSF by describing the autopsy experience at the Massachusetts General Hospital. Design.—We describe the findings in a series of 4 autopsy cases of patients diagnosed with NSF. This report describes the history of renal dysfunction, exposure to gadolinium-containing contrast agents, specific laboratory parameters, and the extent of systemic involvement identified by postmortem examination. Results.—Causes of death included systemic thromboembolic disease (n = 3) and pneumonia (n = 1). Laboratory parameters and type, dose, or timing of gadolinium-containing contrast-agent exposure did not correlate with clinical findings and outcomes. All patients demonstrated cutaneous manifestations of the disease and nephrocalcinosis, with some exhibiting calcification and fibrosis of the dura, thyroid, and heart including the cardiac conduction system, on postmortem examination. Soft tissue calcification was associated with concurrent hyperparathyroidism or high serum parathyroid hormone levels. Conclusions.—Thromboembolic disease can be a significant clinical complication of NSF. Patients with NSF may also develop characteristic histologic features of fibrosis and calcification in multiple organs, with significant morbidity and mortality. This autopsy series highlights the variability of systemic manifestations of NSF.

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::656aaf80c2b9f78756c5346dea42463dTest
https://doi.org/10.5858/133.12.1943Test

المؤلفون: Nancy, McCabe, Alton Brad, Farris, Huiming, Hon, Ryan, Ford, Wendy M, Book

المصدر: Congenital heart disease. 8(5)

مصطلحات موضوعية: Heart Valve Prosthesis Implantation, Male, Pulmonary Valve, Carcinoma, Hepatocellular, Liver Neoplasms, Tetralogy of Fallot, Humans, Tricuspid Valve, Middle Aged, Fontan Procedure

الوصف: Liver fibrosis is a growing concern among adults with congenital heart disease, particularly for those who have undergone a Fontan operation. Liver fibrosis leads to cirrhosis, a precursor of hepatocellular carcinoma. A few cases of hepatocellular carcinoma in patients with prior palliative surgery for congenital heart disease have been identified in the literature. The current case reports the first known case of hepatocellular carcinoma in a 45-year-old male with repaired tetralogy of Fallot.

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=pmid________::689c7838728b9349a701ee23fdb0c468Test
https://pubmed.ncbi.nlm.nih.gov/22897884Test