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G. Andrieux

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Open access Aug 2026

Elevated IL-10 is Linked With the Expansion of T-bethighCD21low B Cells in Patients With Common Variable Immunodeficiency

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. · 0 citations
#gene editing Open access Sep 2026

Targeted insertion of an optimized donor DNA is effective in a humanized mouse model of dominant retinitis pigmentosa.

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. · 0 citations
Open access Aug 2026

Pegtarazimod limits acute graft-versus-host disease mortality and severity in mice and is tolerated in patients.

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. · 0 citations
Open access Jul 2026

CD117 epitope-shielded hematopoietic stem cell transplantation with toxin-free conditioning and in vivo selection ameliorates a β-thalassemia model

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. · 0 citations

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