Zobrazeno 1 - 8
of 8
pro vyhledávání: '"Alisa Dong"'
Autor:
Alisa Dong, Valentina Ghiaccio, Irene Motta, Shuling Guo, Raechel Peralta, Susan M. Freier, Andy Watt, Sagar Damle, Yasuhiro Ikawa, Danuta Jarocha, Maxwell Chappell, Coralea Stephanou, Paola Delbini, Connie Chen, Soteroula Christou, Marina Kleanthous, Kim Smith-Whitley, Deepa Manwani, Carla Casu, Osheiza Abdulmalik, Maria Domenica Cappellini, Stefano Rivella, Laura Breda
Publikováno v:
Haematologica, Vol 106, Iss 5 (2020)
β-thalassemia is a disorder caused by altered hemoglobin protein synthesis and affects individuals worldwide. Severe forms of the disease, left untreated, can result in death before the age of 3 years (1). The standard of care consists of chronic an
Externí odkaz:
https://doaj.org/article/0a9af0f8d129480daf7391c3232c9430
Autor:
Paola Delbini, Maxwell Chappell, Valentina Ghiaccio, Danuta Jarocha, Coralea Stephanou, Maria Domenica Cappellini, Marina Kleanthous, Soteroula Christou, Shuling Guo, Kim Smith-Whitley, Irene Motta, Carla Casu, Alisa Dong, Deepa Manwani, Connie Chen, Susan M. Freier, Stefano Rivella, Andrew T. Watt, Laura Breda, Yasuhiro Ikawa, Raechel Peralta, Osheiza Abdulmalik, Sagar S. Damle
Publikováno v:
Haematologica
β-thalassemia is a disorder caused by altered hemoglobin protein synthesis and affects individuals worldwide. Severe forms of the disease, left untreated, can result in death before the age of 3 years (1). The standard of care consists of chronic an
Autor:
Tobias Raabe, Janet L. Kwiatkowski, Aoife M. Doto, Alisa Dong, Frederic D. Bushman, Charles A. Assenmacher, Enrico Radaelli, John K. Everett, Carla Casu, Danuta Jarocha, Kim Smith-Whitley, Osheiza Abdulmalik, Xiaochuan Shan, Laura Breda, James C. Tarrant, Yasuhiro Ikawa, Ryo Kurita, Virginia Guzikowski, Gwenn Danet-Desnoyers, Naoto Tanaka, Natalie Hoepp, Stefano Rivella, Yukio Nakamura, Valentina Ghiaccio
Publikováno v:
Mol Ther
Ongoing clinical trials for treatment of beta-globinopathies by gene therapy involve the transfer of the beta-globin gene, which requires integration of three to four copies per genome in most target cells. This high proviral load may increase genome
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::cb268911a830997d4bc834d262854d0c
https://europepmc.org/articles/PMC8058492/
https://europepmc.org/articles/PMC8058492/
Autor:
Omar Niss, Monika Asnani, Alisa Dong, Punam Malik, Archana Shrestha, Carolyn Lutzko, Charles T. Quinn, Paritha Arumugam, Sydney Felker, Courtney R Little, Scott Witting, Chris Lo, Jennifer Knight-Madden, Michael Grimley
Publikováno v:
Blood. 138:3970-3970
Introduction: Sickle cell disease (SCD) is a genetic red blood cell (RBC) disorder that causes chronic hemolytic anemia, progressive organ damage, and life-threatening acute complications such as painful vaso-occlusive crises. Allogeneic hematopoieti
Autor:
Soteroula Christou, Alisa Dong, Shuling Guo, Deepa Manwani, Laura Breda, Connie Chen, Yasuhiro Ikawa, Stefano Rivella, Susan M. Freier, Kim Smith-Whitley, Marina Kleanthous, Valentina Ghiaccio, Andrew T. Watt, Carla Casu, Irene Motta, Osheiza Abdulmalik, Raechel Peralta, Maxwell Chappell, Coralea Stephanou, Maria Domenica Cappellini, Paola Delbini
Publikováno v:
Blood. 134:2244-2244
The β thalassemia trait is associated with over 300 mutations in the β-globin gene that lead to reduced (β+ allele) or absent (β0 allele) synthesis of the β globin chain. A subset of these mutations affect the canonic splicing of the β globin m
Publikováno v:
Translational Research. 161:293-306
Hemoglobinopathies are genetic inherited conditions that originate from the lack or malfunction of the hemoglobin (Hb) protein. Sickle cell disease (SCD) and thalassemia are the most common forms of these conditions. The severe anemia combined with c
Autor:
Mathew G. Angelos, Jacopo Baglieri, Carmen Bertoni, Laura Breda, Hildegard Büning, Lawrence Chan, Wenhao Chen, Laurence J.N. Cooper, Alisa Dong, Christopher H. Evans, Charles A. Gersbach, Steven C. Ghivizzani, Saar Gill, Joseph C. Glorioso, William F. Goins, Perry B. Hackett, H. Kirk Hammond, Manu Jain, Michael Kalos, Dan S. Kaufman, Fahad Kidwai, Christopher D. Kontos, Robert A. Kratzke, Robert E. MacLaren, Michelle E. McClements, Federico Mingozzi, Sarah B. Mueller, Jianfang Ning, Manish R. Patel, Michelle Prickett, Samuel D. Rabkin, Stefano Rivella, Paul D. Robbins, Michele Simonato, Timothy K. Starr, Tong Tang, Pratiksha I. Thakore, Jakub Tolar, Lars U. Wahlberg, Christopher E. Walsh, Jie Wu, Aini Xie, Yisheng Yang
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::e2acff10e9747151e502cf79260c8a39
https://doi.org/10.1016/b978-0-12-800563-7.01002-2
https://doi.org/10.1016/b978-0-12-800563-7.01002-2
Hemoglobinopathies are inherited genetic conditions that originate from a lack or malfunction of the adult hemoglobin protein. Thalassemia and other diseases associated with β-globin abnormal amino acid sequences—such as sickle cell disease (SCD)
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::d800843fda972bed2f39c4b3a73e0db8
https://doi.org/10.1016/b978-0-12-800563-7.00012-9
https://doi.org/10.1016/b978-0-12-800563-7.00012-9