Common variable immunodeficiency (CVID) is frequently complicated by autoimmune and inflammatory manifestations associated with profound immune dysregulation (CVIDc), including expansion of T-bethighCD21low B cells (CD21low B cells). Elevated serum IL-10 levels have repeatedly been reported in CVIDc, yet their relation...
Victoria Cousin, A. Graumann, V. Geiger et al.· Journal of Clinical Immunolo...· 0 citations
Retinitis pigmentosa (RP) affects 1 in 3,000 individuals worldwide, with 30%-40% of cases inherited as autosomal dominant (AD). Mutations in RHO (RP4) are the most common cause of ADRP. Because most RHO mutations exert gain-of-function or dominant-negative effects, conventional gene supplementation is insufficient, req...
F. Esposito, Arjun Padmanabhan, M. Rhiel et al.· Cell Reports Medicine· 0 citations
Pegtarazimod reduced aGVHD in mice by suppressing pro inflammatory neutrophil effector functions and preserving enterocyte integrity and the clinical trial data support tolerability of pegtarazimod in aGVHD patients and further studies are needed to determine efficacy.
Verena Holzmüller, Jana Gawron, Ann-Cathrin Burk et al.· Journal of Clinical Investig...· 0 citations
The results suggest that this approach may overcome the reliance on busulfan or other myeloablative conditioning regimens with their associated morbidities, and by enabling toxin-free conditioning and in vivo selection of edited cells, may facilitate clinical implementation of these highly valuable genetic therapies.
Romina Marone, Rosalba Lepore, K. Paschoudi et al.· bioRxiv· 0 citations
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