Results 91 to 100 of about 1,824,389 (266)

Gene Therapy for Hemoglobinopathies [PDF]

Human Gene Therapy, 2018
Gene therapy for β-thalassemia and sickle-cell disease is based on transplantation of genetically corrected, autologous hematopoietic stem cells. Preclinical and clinical studies have shown the safety and efficacy of this therapeutic approach, currently based on lentiviral vectors to transfer a β-globin gene under the transcriptional control of ...
Cavazzana M., Mavilio F.
openaire   +3 more sources

Identifying microRNA determinants of human myelopoiesis

Scientific Reports, 2018
Myelopoiesis involves differentiation of hematopoietic stem cells to cellular populations that are restricted in their self-renewal capacity, beginning with the common myeloid progenitor (CMP) and leading to mature cells including monocytes and ...
Megha Rajasekhar, Ulf Schmitz, Stephane Flamant, Justin J.-L. Wong, Charles G. Bailey, William Ritchie, Jeff Holst, John E. J. Rasko   +7 more
doaj   +1 more source

Gene therapy [PDF]

Nature, 1993
Mavilio F., Bordignon C.
  +8 more sources

Intron retention is regulated by altered MeCP2-mediated splicing factor recruitment

Nature Communications, 2017
Intron retention is a conserved mechanism that controls gene expression but its regulation is poorly understood. Here, the authors provide evidence that DNA methylation regulates intron retention and find reduced MeCP2 occupancy and splicing factor ...
Justin J. -L. Wong, Dadi Gao, Trung V. Nguyen, Chau-To Kwok, Michelle van Geldermalsen, Rob Middleton, Natalia Pinello, Annora Thoeng, Rajini Nagarajah, Jeff Holst, William Ritchie, John E. J. Rasko   +11 more
doaj   +1 more source

In Utero Transplantation of Placenta-Derived Mesenchymal Stromal Cells for Potential Fetal Treatment of Hemophilia A. [PDF]

, 2018
Hemophilia A (HA) is an X-linked recessive disorder caused by mutations in the factor VIII ( FVIII) gene leading to deficient blood coagulation. The current standard of care is frequent infusions of plasma-derived FVIII or recombinant B-domain-deleted ...
Farmer, Diana, Gao, Kewa, Kumar, Priyadarsini, Lankford, Lee, Pivetti, Christopher, Wang, Aijun, Wang, Chuwang   +6 more
core   +1 more source

ProteinCoLoc streamlines Bayesian analysis of colocalization in microscopic images

Scientific Reports
Colocalization, the spatial overlap of molecular entities, is often key to support their involvement in common functions. Existing colocalization tools, however, face limitations, particularly because of their basic statistical analysis and their low ...
Manuel Seefelder, Stefan Kochanek, Fabrice A. C. Klein   +2 more
doaj   +1 more source

Discovery of cancer common and specific driver gene sets [PDF]

arXiv, 2016
Cancer is known as a disease mainly caused by gene alterations. Discovery of mutated driver pathways or gene sets is becoming an important step to understand molecular mechanisms of carcinogenesis. However, systematically investigating commonalities and specificities of driver gene sets among multiple cancer types is still a great challenge, but this ...
arxiv  

Gene Therapy – Potential, Pros, Cons and Ethics [PDF]

, 2002
Genetic technology poses risks along with its rewards, just as any technology has in the past. To stop its development and forfeit the benefits gene therapy could offer would be a far greater mistake than forging ahead could ever be.
Rao Nanjunda, Mr Ananth
core  

The antiproliferative ELF2 isoform, ELF2B, induces apoptosis in vitro and perturbs early lymphocytic development in vivo

Journal of Hematology & Oncology, 2017
Background ELF2 (E74-like factor 2) also known as NERF (new Ets-related factor), a member of the Ets family of transcription factors, regulates genes important in B and T cell development, cell cycle progression, and angiogenesis. Conserved ELF2 isoforms,
Fiona H. X. Guan, Charles G. Bailey, Cynthia Metierre, Patrick O’Young, Dadi Gao, Teh Liane Khoo, Jeff Holst, John E. J. Rasko   +7 more
doaj   +1 more source

CD28.OX40 co-stimulatory combination is associated with long in vivo persistence and high activity of CAR.CD30 T-cells

Haematologica, 2020
The prognosis of many patients with chemotherapy-refractory or multiply relapsed CD30+ non-Hodgkin Lymphoma (NHL) or Hodgkin lymphoma (HL) still remains poor, and novel therapeutic approaches are warranted to address this unmet clinical need.
Marika Guercio, Domenico Orlando, Stefano Di Cecca, Matilde Sinibaldi, Iolanda Boffa, Simona Caruso, Zeinab Abbaszadeh, Antonio Camera, Biancamaria Cembrola, Katia Bovetti, Simona Manni, Ignazio Caruana, Roselia Ciccone, Francesca Del Bufalo, Pietro Merli, Luciana Vinti, Katia Girardi, Annalisa Ruggeri, Cristiano De Stefanis, Marco Pezzullo, Ezio Giorda, Marco Scarsella, Rita De Vito, Sabina Barresi, Andrea Ciolfi, Marco Tartaglia, Lorenzo Moretta, Franco Locatelli, Concetta Quintarelli, Biagio De Angelis   +29 more
doaj   +1 more source