Bridget Yates
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View article: Comparative Analysis of CRISPR-Cas9, lentiviral transduction, and base editing for sickle cell disease in a murine model
Comparative Analysis of CRISPR-Cas9, lentiviral transduction, and base editing for sickle cell disease in a murine model Open
Sickle Cell Disease (SCD) is a red blood cell disorder caused by a mutation in the β-globin gene, leading to sickle hemoglobin (HbS) polymerization under low oxygen conditions. CRISPR-Cas9 editing and lentiviral transduction have shown pro…
View article: Prophylactic Prednisolone Promotes AAV5 Hepatocyte Transduction Through the Novel Mechanism of AAV5 Coreceptor Platelet-Derived Growth Factor Receptor Alpha Upregulation and Innate Immune Suppression
Prophylactic Prednisolone Promotes AAV5 Hepatocyte Transduction Through the Novel Mechanism of AAV5 Coreceptor Platelet-Derived Growth Factor Receptor Alpha Upregulation and Innate Immune Suppression Open
Adeno-associated virus (AAV) vectors are used to deliver therapeutic transgenes, but host immune responses may interfere with transduction and transgene expression. We evaluated prophylactic corticosteroid treatment on AAV5-mediated expres…
View article: 2334. The effectiveness of bivalent COVID-19 booster among patients with hematologic malignancies
2334. The effectiveness of bivalent COVID-19 booster among patients with hematologic malignancies Open
Background Patients with hematologic malignancies remain at higher risk for developing severe SARS-CoV-2 infections and have reduced immune responses to vaccines. Methods A prospective observational study was conducted at Brigham and Women…
View article: 2707. Complications of Babesiosis in Immunocompromised and Asplenic/Hyposplenic Patients
2707. Complications of Babesiosis in Immunocompromised and Asplenic/Hyposplenic Patients Open
Background Babesiosis is a tick-borne disease frequently encountered in the region of New England in the US, with increasing incidence and complications ranging from hemolysis to end-organ damage and death. Immunocompromised (IC) patients …
View article: Young mice administered adult doses of AAV5-hFVIII-SQ achieve therapeutic factor VIII expression into adulthood
Young mice administered adult doses of AAV5-hFVIII-SQ achieve therapeutic factor VIII expression into adulthood Open
Valoctocogene roxaparvovec (AAV5-hFVIII-SQ) gene transfer provided reduced bleeding for adult clinical trial participants with severe hemophilia A. However, pediatric outcomes are unknown. Using a mouse model of hemophilia A, we investigat…
View article: Molecular analysis of AAV5-hFVIII-SQ vector-genome-processing kinetics in transduced mouse and nonhuman primate livers
Molecular analysis of AAV5-hFVIII-SQ vector-genome-processing kinetics in transduced mouse and nonhuman primate livers Open
Valoctocogene roxaparvovec (AAV5-hFVIII-SQ) is an adeno-associated virus serotype 5 (AAV5)-based gene therapy vector containing a B-domain-deleted human coagulation factor VIII (hFVIII) gene controlled by a liver-selective promoter. AAV5-h…
View article: Strategy to detect pre-existing immunity to AAV gene therapy
Strategy to detect pre-existing immunity to AAV gene therapy Open
Gene therapy may offer a new treatment option, particularly for patients with severe hemophilia, based on recent research. However, individuals with pre-existing immunity to adeno-associated viruses (AAVs) may be less likely to benefit fro…
View article: Clearance of Heparan Sulfate and Attenuation of CNS Pathology by Intracerebroventricular BMN 250 in Sanfilippo Type B Mice
Clearance of Heparan Sulfate and Attenuation of CNS Pathology by Intracerebroventricular BMN 250 in Sanfilippo Type B Mice Open
Sanfilippo syndrome type B (mucopolysaccharidosis IIIB), caused by inherited deficiency of α-N-acetylglucosaminidase (NAGLU), required for lysosomal degradation of heparan sulfate (HS), is a pediatric neurodegenerative disorder with no app…