Zobrazeno 1 - 10
of 61
pro vyhledávání: '"Harry Malech"'
Severe combined immunodeficiency: improved survival leading to detection of underlying liver disease
Autor:
Anusha Vittal, Nehna Abdul Majeed, Elizabeth Garabedian, Jamie Marko, David E Kleiner, Rob Sokolic, Fabio Candotti, Harry Malech, Theo Heller, Christopher Koh
Publikováno v:
BMC Gastroenterology, Vol 23, Iss 1, Pp 1-8 (2023)
Abstract Background Adenosine deaminase deficiency (ADA) is an autosomal recessive disorder leading to severe combined immunodeficiency (SCID). It is characterized patho-physiologically by intracellular accumulation of toxic products affecting lympho
Externí odkaz:
https://doaj.org/article/256b0a5a135547408f0e2ed934e6b8d6
Autor:
Mara Pavel-Dinu, Volker Wiebking, Beruh T. Dejene, Waracharee Srifa, Sruthi Mantri, Carmencita E. Nicolas, Ciaran Lee, Gang Bao, Eric J. Kildebeck, Niraj Punjya, Camille Sindhu, Matthew A. Inlay, Nivedita Saxena, Suk See DeRavin, Harry Malech, Maria Grazia Roncarolo, Kenneth I. Weinberg, Matthew H. Porteus
Publikováno v:
Nature Communications, Vol 10, Iss 1, Pp 1-15 (2019)
Gene correction in hematopoietic stem cells could be a powerful way to treat monogenic diseases of the blood and immune system. Here the authors develop a strategy using CRISPR-Cas9 and an aAdeno-Associated vVirus(AAV)-delivered IL2RG cDNA to correct
Externí odkaz:
https://doaj.org/article/66cf921ac2ec4178bebe47ca77ff7c87
Autor:
Mara Pavel-Dinu, Volker Wiebking, Beruh T. Dejene, Waracharee Srifa, Sruthi Mantri, Carmencita E. Nicolas, Ciaran Lee, Gang Bao, Eric J. Kildebeck, Niraj Punjya, Camille Sindhu, Matthew A. Inlay, Nivedita Saxena, Suk See DeRavin, Harry Malech, Maria Grazia Roncarolo, Kenneth I. Weinberg, Matthew H. Porteus
Publikováno v:
Nature Communications, Vol 10, Iss 1, Pp 1-1 (2019)
An amendment to this paper has been published and can be accessed via a link at the top of the paper.
Externí odkaz:
https://doaj.org/article/91a0613f85d54ca698128b995ca0d27f
Autor:
Natalia S. Pripuzova, Melkamu Getie-Kebtie, Christopher Grunseich, Colin Sweeney, Harry Malech, Michail A. Alterman
Publikováno v:
Stem Cell Research, Vol 14, Iss 3, Pp 323-338 (2015)
The emergence of new methods for reprogramming of adult somatic cells into induced pluripotent stem cells (iPSC) led to the development of new approaches in drug discovery and regenerative medicine. Investigation of the molecular mechanisms underlyin
Externí odkaz:
https://doaj.org/article/9b213351657a419c9e1c9291a9d38a31
Autor:
Karlie Jones, Angela Ballesteros, Margaret Mentink-Kane, James Warren, Shemona Rattila, Harry Malech, Elizabeth Kang, Gabriela Dveksler
Publikováno v:
PLoS ONE, Vol 11, Iss 7, p e0158050 (2016)
The pregnancy-specific glycoproteins (PSGs) are a family of proteins secreted by the syncytiotrophoblast of the placenta and are the most abundant trophoblastic proteins in maternal blood during the third trimester. The human PSG family consists of 1
Externí odkaz:
https://doaj.org/article/d6de079d1f2447da9cad73dbd11c5fe9
Autor:
Morgan N. Similuk, Jia Yan, Rajarshi Ghosh, Andrew J. Oler, Luis M. Franco, Michael R. Setzer, Michael Kamen, Colleen Jodarski, Thomas DiMaggio, Joie Davis, Rachel Gore, Leila Jamal, Adrienne Borges, Nicole Gentile, Julie Niemela, Chenery Lowe, Kathleen Jevtich, Yunting Yu, Haley Hullfish, Amy P. Hsu, Celine Hong, Patricia Littel, Bryce A. Seifert, Joshua Milner, Jennifer J. Johnston, Xi Cheng, Zhiwen Li, Daniel Veltri, Ke Huang, Krishnaveni Kaladi, Jason Barnett, Lingwen Zhang, Nikita Vlasenko, Yongjie Fan, Eric Karlins, Satishkumar Ranganathan Ganakammal, Robert Gilmore, Emily Tran, Alvin Yun, Joseph Mackey, Svetlana Yazhuk, Justin Lack, Vasudev Kuram, Wenjia Cao, Susan Huse, Karen Frank, Gary Fahle, Sergio Rosenzweig, Yan Su, SuJin Hwang, Weimin Bi, John Bennett, Ian A. Myles, Suk See De Ravin, Ivan Fuss, Warren Strober, Bibiana Bielekova, Adriana Almeida de Jesus, Raphaela Goldbach-Mansky, Peter Williamson, Kelly Kumar, Caeden Dempsy, Pamela Frischmeyer-Guerrerio, Robin Fisch, Hyejeong Bolan, Dean D. Metcalfe, Hirsh Komarow, Melody Carter, Kirk M. Druey, Irini Sereti, Lesia Dropulic, Amy D. Klion, Paneez Khoury, Elise M. O' Connell, Nicole C. Holland-Thomas, Thomas Brown, David H. McDermott, Philip M. Murphy, Vanessa Bundy, Michael D. Keller, Christine Peng, Helen Kim, Stephanie Norman, Ottavia M. Delmonte, Elizabeth Kang, Helen C. Su, Harry Malech, Alexandra Freeman, Christa Zerbe, Gulbu Uzel, Jenna R.E. Bergerson, V. Koneti Rao, Kenneth N. Olivier, Jonathan J. Lyons, Andrea Lisco, Jeffrey I. Cohen, Michail S. Lionakis, Leslie G. Biesecker, Sandhya Xirasagar, Luigi D. Notarangelo, Steven M. Holland, Magdalena A. Walkiewicz
Publikováno v:
J Allergy Clin Immunol
BACKGROUND: Prospective genetic evaluation of patients at our referral research hospital presents clinical research challenges. OBJECTIVE: This study sought not only a single-gene explanation for participants’ immune-related presentations, but view
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::c8f8a28d08c3a4960c10b7fc3d195a47
https://doi.org/10.1016/j.jaci.2022.06.009
https://doi.org/10.1016/j.jaci.2022.06.009
Autor:
Quentin Riller, Jacques Fourgeaud, Julie Bruneau, Suk See De Ravin, Grace Smith, Mathieu Fusaro, Samy Meriem, Aude Magerus, Marine Luka, Ghaith Abdessalem, Ludovic Lhermitte, Anne Jamet, Emmanuelle Six, Alessandra Magnani, Martin Castelle, Romain Lévy, Mathilde M. Lecuit, Benjamin Fournier, Sarah Winter, Michaela Semeraro, Graziella Pinto, Hanène Abid, Nizar Mahlaoui, Nathalie Cheikh, Benoit Florkin, Pierre Frange, Eric Jeziorski, Felipe Suarez, Françoise Sarrot-Reynauld, Dalila Nouar, Dominique Debray, Florence Lacaille, Capucine Picard, Philippe Pérot, Béatrice Regnault, Nicolas Da Rocha, Camille de Cevins, Laure Delage, Brieuc P. Pérot, Angélique Vinit, Francesco Carbone, Camille Brunaud, Manon Marchais, Marie-Claude Stolzenberg, Vahid Asnafi, Thierry Molina, Frédéric Rieux-Laucat, Luigi D. Notarangelo, Stefania Pittaluga, Jean Philippe Jais, Despina Moshous, Stephane Blanche, Harry Malech, Marc Eloit, Marina Cavazzana, Alain Fischer, Mickaël M. Ménager, Bénédicte Neven
Publikováno v:
Journal of Allergy and Clinical Immunology.
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::32b7b542cd56d980c7542731c4a12b5d
https://doi.org/10.1016/j.jaci.2022.12.822
https://doi.org/10.1016/j.jaci.2022.12.822
Severe Combined Immunodeficiency: Improved survival leading to detection of underlying liver disease
Autor:
Anusha Vittal, Nehna Abdul Majeed, Elizabeth Garabedian, Jamie Marko, David E Kleiner, Rob Sokolic, Fabio Candotti, Harry Malech, Theo Heller, Christopher Koh
Background Adenosine deaminase deficiency (ADA) is an autosomal recessive disorder leading to severe combined immunodeficiency (SCID). It is characterized patho-physiologically by intracellular accumulation of toxic products affecting lymphocytes. Ot
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::2e36948ae56ed19ce6998ec51532b5e8
https://doi.org/10.21203/rs.3.rs-1915188/v1
https://doi.org/10.21203/rs.3.rs-1915188/v1
Autor:
Alexandre Villard, Christa Zerbe, Colin Sweeney, Suk See DeRavin, Harry Malech, Steven Holland, Emilia Falcone
Publikováno v:
Clinical Immunology. 250:109391
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::6aab856d2e2915305b54ddcae4bc50aa
https://doi.org/10.1016/j.clim.2023.109391
https://doi.org/10.1016/j.clim.2023.109391
Publikováno v:
Clinical Immunology. 250:109543
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::d7c9fd5fd3c5d70e9ce83325276f67f2
https://doi.org/10.1016/j.clim.2023.109543
https://doi.org/10.1016/j.clim.2023.109543