Zobrazeno 1 - 10
of 13
pro vyhledávání: '"Christopher T. Lux"'
Autor:
Sowmya Pattabhi, Samantha N. Lotti, Mason P. Berger, Swati Singh, Christopher T. Lux, Kyle Jacoby, Calvin Lee, Olivier Negre, Andrew M. Scharenberg, David J. Rawlings
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 17, Iss , Pp 277-288 (2019)
Gene editing following designer nuclease cleavage in the presence of a DNA donor template can revert mutations in disease-causing genes. For optimal benefit, reversion of the point mutation in HBB leading to sickle cell disease (SCD) would permit pre
Externí odkaz:
https://doaj.org/article/5c5d0cc4529c44adb9273c8f4a9f26d9
Autor:
Christopher T. Lux, Sowmya Pattabhi, Mason Berger, Cynthia Nourigat, David A. Flowers, Olivier Negre, Olivier Humbert, Julia G. Yang, Calvin Lee, Kyle Jacoby, Irwin Bernstein, Hans-Peter Kiem, Andrew Scharenberg, David J. Rawlings
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 12, Iss , Pp 175-183 (2019)
Elements within the γ-hemoglobin promoters (HBG1 and HBG2) function to bind transcription complexes that mediate repression of fetal hemoglobin expression. Sickle cell disease (SCD) subjects with a 13-bp deletion in the HBG1 promoter exhibit a clini
Externí odkaz:
https://doaj.org/article/d1669924b97e44a4beafad9d2ebc6f23
Autor:
David J. Rawlings, Samantha N. Lotti, Olivier Negre, Kyle Jacoby, Swati Singh, Sowmya Pattabhi, Andrew M. Scharenberg, Calvin Lee, Christopher T. Lux, Mason P. Berger
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 17, Iss, Pp 277-288 (2019)
Molecular Therapy. Nucleic Acids
Molecular Therapy. Nucleic Acids
Gene editing following designer nuclease cleavage in the presence of a DNA donor template can revert mutations in disease-causing genes. For optimal benefit, reversion of the point mutation in HBB leading to sickle cell disease (SCD) would permit pre
Autor:
Irwin D. Bernstein, Julia G. Yang, Andrew M. Scharenberg, Christopher T. Lux, Olivier Negre, Sowmya Pattabhi, David A. Flowers, Kyle Jacoby, Mason P. Berger, Hans-Peter Kiem, Calvin Lee, Cynthia Nourigat, Olivier Humbert, David J. Rawlings
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 12, Iss, Pp 175-183 (2019)
Molecular Therapy. Methods & Clinical Development
Molecular Therapy. Methods & Clinical Development
Elements within the γ-hemoglobin promoters (HBG1 and HBG2) function to bind transcription complexes that mediate repression of fetal hemoglobin expression. Sickle cell disease (SCD) subjects with a 13-bp deletion in the HBG1 promoter exhibit a clini
Autor:
Olivier Negre, Christopher W. Peterson, Christopher T. Lux, Sowmya Reddy, Ray R. Carrillo, Clare Samuelson, Anai M. Perez, Sowmya Pattabhi, Stefan Radtke, Ciaran M. Lee, David J. Rawlings, Jennifer E. Adair, Gang Bao, Lauren E Schefter, Olivier Humbert, Hans-Peter Kiem, Andrew M. Scharenberg
Publikováno v:
Sci Transl Med
Reactivation of fetal hemoglobin (HbF) is being pursued as a treatment strategy for hemoglobinopathies. Here, we evaluated the therapeutic potential of hematopoietic stem and progenitor cells (HSPCs) edited with the CRISPR/Cas9 nuclease platform to r
Autor:
Christopher W. Peterson, Ciaran M. Lee, Olivier Negre, Christopher T. Lux, David J. Rawlings, Gang Bao, Anai M. Perez, Lauren E Schefter, Hans-Peter Kiem, Sowmya Reddy, Ray R. Carillo, Olivier Humbert, Andrew M. Scharenberg, Stefan Radtke, Jennifer E. Adair, Sowmya Pattabhi
Publikováno v:
Blood. 132:806-806
Beta-thalassemia and sickle cell disease are monogenic disorders that are currently treated by allogeneic bone marrow (BM) transplantation although the challenges of finding a suitable matched-donor and the risk of graft vs host disease have limited
Autor:
Christopher T. Lux, Mervin C. Yoder
The mammalian yolk sac is known to play a prominent role in emergence of the hematopoietic system. The extent of this contribution has been a subject of debate in recent years largely due to effects of the early circulation that obscures the site of
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::245b6b02a16f411ff3c2d214474df6a0
https://europepmc.org/articles/PMC2944404/
https://europepmc.org/articles/PMC2944404/
Autor:
James Palis, Kathleen E. McGrath, Momoko Yoshimoto, Simon J. Conway, Christopher T. Lux, Mervin C. Yoder
Publikováno v:
Blood. 111(7)
The relative contribution of yolk sac (YS)–derived cells to the circulating definitive hematopoietic progenitor cell (HPC) pool that seeds the fetal liver remains controversial due to the presence of systemic circulation and the onset of hematopoie
Publikováno v:
Molecular Therapy. 23:S262
Hemoglobinopathies are common and morbid inherited conditions caused by mutations in hemoglobin genes that generate pathologic globin molecules or reduced expression of wild type hemoglobin. A subset of individuals with hemoglobinopathy-associated be
Autor:
Mervin C. Yoder, Christos Gekas, Katrin E. Rhodes, Cameron S. Francis, Yanling Wang, Simon J. Conway, Hanna K. A. Mikkola, Stuart H. Orkin, Christopher T. Lux
Publikováno v:
Blood. 110:1258-1258
The placenta was recently unveiled as an important hematopoietic organ, harboring a large pool of HSCs during midgestation. Yet, it has not been defined whether the placenta can generate HSCs de novo. By using the Runx1-LacZ and Ncx1 knockout mouse m