Publications by authors named "P K Morrow"
Article Synopsis
- Exagamglogene autotemcel (exa-cel) is a nonviral cell therapy utilizing CRISPR-Cas9 gene editing to increase fetal hemoglobin production in patients with sickle cell disease.
- A phase 3 study involved 44 patients aged 12 to 35 with a history of severe vaso-occlusive crises; patients received edited stem cells after myeloablative conditioning.
- Results showed 97% of patients were free from vaso-occlusive crises and 100% avoided hospitalization for over 12 months, with a safety profile similar to standard treatments.
Article Synopsis
- Exagamglogene autotemcel (exa-cel) is a novel cell therapy using CRISPR-Cas9 gene editing to boost fetal hemoglobin production in patients with transfusion-dependent β-thalassemia.
- In a phase 3 study, 52 patients aged 12 to 35 underwent treatment with exa-cel after myeloablative conditioning, and 91% achieved transfusion independence for at least 12 months.
- The therapy showed promising results with high mean total and fetal hemoglobin levels, a favorable safety profile, and no serious adverse events like deaths or cancers reported during the follow-up period.
Therapeutic approaches for clear cell renal cell carcinoma (ccRCC) remain limited; however, chimeric antigen receptor (CAR) T-cell therapies may offer novel treatment options. CTX130, an allogeneic CD70-targeting CAR T-cell product, was developed for the treatment of advanced or refractory ccRCC. We report that CTX130 showed favorable preclinical proliferation and cytotoxicity profiles and completely regressed RCC xenograft tumors.
View Article and Find Full Text PDFIntroduction: The physical impact of alopecia areata (AA) is visible, but the psychological and social consequences and emotional burden are often underrecognized.
Methods: In this cross-sectional study, 547 participants recruited via the National Alopecia Areata Foundation completed a survey encompassing demographics; AA illness characteristics; and five patient-reported outcome measures on anxiety and depression, perceived stress, psychological illness impact, stigma, and quality of life (QoL). Differences in disease severity subgroups were assessed via analysis of variance (ANOVA) and t tests.