Scott Haskett
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View article: 155 CD5 knockout enhances the potency of multiplex base-edited allogeneic anti-CD5 CAR T-cell therapy for the treatment of T-cell malignancies
155 CD5 knockout enhances the potency of multiplex base-edited allogeneic anti-CD5 CAR T-cell therapy for the treatment of T-cell malignancies Open
Background T-cell lymphomas and leukemias are a class of diseases lacking durable effective therapies, where median survival for patients suffering from relapsed/refractory disease is often measured in months. Translation of B-cell targeti…
View article: PS1518 GENOME EDITING OF HBG1/2 PROMOTER LEADS TO ROBUST HBF INDUCTION IN VIVO, WHILE EDITING OF BCL11A ERYTHROID ENHANCER RESULTS IN ERYTHROID DEFECTS
PS1518 GENOME EDITING OF HBG1/2 PROMOTER LEADS TO ROBUST HBF INDUCTION IN VIVO, WHILE EDITING OF BCL11A ERYTHROID ENHANCER RESULTS IN ERYTHROID DEFECTS Open
Background: Induction of fetal hemoglobin (HbF) is an attractive approach to ameliorate disease symptoms in beta hemoglobinopathies. Gene editing by zinc finger or CRISPR‐Cas nucleases to disrupt BCL11A expression, a known mediator of feta…
View article: Somatic Gene Editing of <i>GUCY2D</i> by AAV-CRISPR/Cas9 Alters Retinal Structure and Function in Mouse and Macaque
Somatic Gene Editing of <i>GUCY2D</i> by AAV-CRISPR/Cas9 Alters Retinal Structure and Function in Mouse and Macaque Open
Mutations in GUCY2D, the gene encoding retinal guanylate cyclase-1 (retGC1), are the leading cause of autosomal dominant cone-rod dystrophy (CORD6). Significant progress toward clinical application of gene replacement therapy for Le…
View article: Efficient in vivo editing of CEP290 IVS26 by EDIT-101 as a novel therapeutic for treatment of Leber Congenital Amaurosis 10
Efficient in vivo editing of CEP290 IVS26 by EDIT-101 as a novel therapeutic for treatment of Leber Congenital Amaurosis 10 Open
View article: RNA-Seq and CyTOF immuno-profiling of regenerating lacrimal glands identifies a novel subset of cells expressing muscle-related proteins
RNA-Seq and CyTOF immuno-profiling of regenerating lacrimal glands identifies a novel subset of cells expressing muscle-related proteins Open
The purpose of the present studies was to use CyTOF and RNA-Seq technologies to identify cells and genes involved in lacrimal gland repair that could be targeted to treat diseases of lacrimal gland dysfunction. Lacrimal glands of female BA…
View article: Clinical relevance of RORγ positive and negative subsets of CD161<sup>+</sup>CD4<sup>+</sup>T cells in primary Sjögren’s syndrome
Clinical relevance of RORγ positive and negative subsets of CD161<sup>+</sup>CD4<sup>+</sup>T cells in primary Sjögren’s syndrome Open
In addition to pointing to CD161 as a marker of a pathogenic subset of CD4+ T cells in pSS patients, our data indicate that even though the RORγ+ (Th17) CD161+ subset might contribute to humoral manifestati…
View article: Peptide-MHC-I from Endogenous Antigen Outnumber Those from Exogenous Antigen, Irrespective of APC Phenotype or Activation
Peptide-MHC-I from Endogenous Antigen Outnumber Those from Exogenous Antigen, Irrespective of APC Phenotype or Activation Open
Naïve anti-viral CD8+ T cells (TCD8+) are activated by the presence of peptide-MHC Class I complexes (pMHC-I) on the surface of professional antigen presenting cells (pAPC). Increasing the number of pMHC-I in vivo can increase the number o…