Kimberly Gilmour

A phase 1 feasibility trial of BE-CAR33, an “off-the-shelf” base-edited CAR33 T cell therapy for acute myeloid leukemia

Christos Georgiadis, Robert Chiesa, Hebatalla Rashed, Bethany K. Hughes et autres

Chimeric antigen receptor (CAR) T cell therapy for acute myeloid leukemia (AML) is constrained by antigen heterogeneity and shared expression with healthy compartments, and there are often challenges in obtaining autologous T cells from heavily pretreated patients. To address these challenges, we …

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Autologous Ex Vivo Lentiviral Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I Provides Sustained Efficacy with a Favorable Safety Profile

Claire Booth, Julián Sevilla, Elena Almarza, Caroline Y. Kuo et autres

Background Severe leukocyte adhesion deficiency-I (LAD-I) results from biallelic deleterious ITGB2 variants leading to deficient/defective CD18 leukocyte expression and impaired endothelial adhesion and extravasation. Children with <2% of normal CD18 neutrophil expression experience recurrent, life-threatening bacterial and fungal infections, and extensive mortality. …

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Assessment of TREC-Based NBS SCID Reporting Practices for Harmonization of Results and Interpretation: A Global Survey

Jahnavi Aluri, Amy Gaviglio, Isaac Kistler, DIANNE R. WEBSTER et autres

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Beyond survival: Multisystem long-term outcomes following HSCT in chronic granulomatous disease

Valentina Guarnieri, Jesmeen Maimaris, Sohilla Lotfy, Elizabeth J Rivers et autres

Data on late complications after hematopoietic stem cell transplantation (HSCT) in children with chronic granulomatous disease (CGD) are limited. We retrospectively analyzed 42 pediatric CGD patients who survived >2 years post-HSCT (1994–2020) at Great Ormond Street Hospital. X-linked CGD accounted for 69%. …

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(667) Antibody Response to Sars-Cov-2 Vaccination in Adolescents Following Solid Organ Transplantation

R. Brugha, Eban Davis, N. Low, E. O'Connor et autres

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