Sofia E. Luna
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View article: Engineering hematopoietic stem and progenitor cells to generate red blood cells as viral traps against HIV-1
Engineering hematopoietic stem and progenitor cells to generate red blood cells as viral traps against HIV-1 Open
Canonical HIV-1 entry into target cells depends on binding to CD4 as a primary receptor. Because of this, use of the CD4 receptor as a viral trap (a decoy receptor used to prevent infection of target cells) is a promising strategy for the …
View article: Multilayered HIV-1 resistance in HSPCs through CCR5 Knockout and B cell secretion of HIV-inhibiting antibodies
Multilayered HIV-1 resistance in HSPCs through CCR5 Knockout and B cell secretion of HIV-inhibiting antibodies Open
Allogeneic transplantation of CCR5 null hematopoietic stem and progenitor cells (HSPCs) is the only known cure for HIV-1 infection. However, this treatment is limited because of the rarity of CCR5-null matched donors, the morbidities assoc…
View article: Engineering synthetic signaling receptors to enable erythropoietin-free erythropoiesis
Engineering synthetic signaling receptors to enable erythropoietin-free erythropoiesis Open
Blood transfusion plays a vital role in modern medicine, but frequent shortages occur. Ex vivo manufacturing of red blood cells (RBCs) from universal donor cells offers a potential solution, yet the high cost of recombinant cytokines remai…
View article: Engineering synthetic signaling receptors to enable erythropoietin-free erythropoiesis
Engineering synthetic signaling receptors to enable erythropoietin-free erythropoiesis Open
Blood transfusion plays a vital role in modern medicine. However, availability is contingent on donated blood, and frequent shortages pose a significant healthcare challenge. Ex vivo manufacturing of red blood cells (RBCs) derived from uni…
View article: Enhancement of erythropoietic output by Cas9-mediated insertion of a natural variant in haematopoietic stem and progenitor cells
Enhancement of erythropoietic output by Cas9-mediated insertion of a natural variant in haematopoietic stem and progenitor cells Open
Some gene polymorphisms can lead to monogenic diseases, whereas other polymorphisms may confer beneficial traits. A well-characterized example is congenital erythrocytosis—the non-pathogenic hyper-production of red blood cells—that is caus…
View article: Engineering inducible signaling receptors to enable erythropoietin-free erythropoiesis
Engineering inducible signaling receptors to enable erythropoietin-free erythropoiesis Open
Blood transfusion plays a vital role in modern medicine. However, availability is contingent on donated blood, and frequent shortages pose a significant healthcare challenge. Ex vivo manufacturing of red blood cells (RBCs) derived from uni…
View article: Engineering inducible signaling receptors to enable erythropoietin-free erythropoiesis
Engineering inducible signaling receptors to enable erythropoietin-free erythropoiesis Open
Blood transfusion plays a vital role in modern medicine. However, availability is contingent on donated blood, and frequent shortages pose a significant healthcare challenge. Ex vivo manufacturing of red blood cells (RBCs) derived from uni…
View article: Combining Cell-Intrinsic and -Extrinsic Resistance to HIV-1 By Engineering Hematopoietic Stem Cells for CCR5 Knockout and B Cell Secretion of Therapeutic Antibodies
Combining Cell-Intrinsic and -Extrinsic Resistance to HIV-1 By Engineering Hematopoietic Stem Cells for CCR5 Knockout and B Cell Secretion of Therapeutic Antibodies Open
Autologous transplantation of CCR5 null hematopoietic stem and progenitor cells (HSPCs) is the only known cure for HIV-1 infection. However, this treatment is limited because of the rarity of CCR5 -null matched donors, the morbidities asso…
View article: Using human genetics to develop strategies to increase erythropoietic output from genome-edited hematopoietic stem and progenitor cells
Using human genetics to develop strategies to increase erythropoietic output from genome-edited hematopoietic stem and progenitor cells Open
Human genetic polymorphisms result in a diversity of phenotypes. Some sequences are pathologic and lead to monogenic diseases, while others may confer beneficial traits. Genome editing is a powerful tool to recreate genotypes found in the …
View article: High-efficiency transgene integration by homology-directed repair in human primary cells using DNA-PKcs inhibition
High-efficiency transgene integration by homology-directed repair in human primary cells using DNA-PKcs inhibition Open
Therapeutic applications of nuclease-based genome editing would benefit from improved methods for transgene integration via homology-directed repair (HDR). To improve HDR efficiency, we screened six small-molecule inhibitors of DNA-depende…
View article: A phase 1/2 open label nonrandomized clinical trial of intravenous 2-hydroxypropyl-β-cyclodextrin for acute liver disease in infants with Niemann-Pick C1
A phase 1/2 open label nonrandomized clinical trial of intravenous 2-hydroxypropyl-β-cyclodextrin for acute liver disease in infants with Niemann-Pick C1 Open
Intravenous 2HPBCD was tolerated in three infants with liver disease due to NPC.
View article: Elucidating the Role of a Lysyl Oxidase Polymorphism in Risk for Coronary Atery Disease
Elucidating the Role of a Lysyl Oxidase Polymorphism in Risk for Coronary Atery Disease Open
From the Washington University Office of Undergraduate Research Digest (WUURD), Vol. 13, 05-01-2018. Published by the Office of Undergraduate Research. Joy Zalis Kiefer, Director of Undergraduate Research and Associate Dean in the College …