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#gene editing Review Aug 2026

Translating CRISPR to Practice in the Clinic: Transformative Therapy in Hemoglobinopathies and Emerging Applications in Malignancies.

Hemoglobinopathies provide the first clinically validated delivery model for CRISPR therapeutics, and offers a donor-independent alternative to allogeneic hematopoietic stem-cell transplantation without graft rejection or graft-versus-host disease.

Miza Salim Hammoud, Rabi Hanna · 0 citations
#gene editing Review Open access Aug 2026

Unlocking the Curative Potential of Gene Transfer and Editing for Hematopoietic Disorders.

Recent developments and refinements in gene transfer and editing technologies for HSPCs are reviewed, while also discussing the critical limitations and hurdles to clinical translation, as recently presented at the New Investigator Committee Gene Therapy webinar.

Kohei Shiroshita, A. Stolz, C. Malouf et al. · 0 citations
#gene editing Review Open access Aug 2026

Epigenetic strategies for fetal hemoglobin induction in sickle cell disease.

This review discusses medicinal chemistry approaches primarily targeting HDAC1/2, LSD1, and DNMT1, with emphasis on inhibitor classes, binding mechanisms, structural features, preclinical evidence, and translational limitations, and how a better understanding of γ-globin repression may guide the development of safer an...

Mateus Mello de Souza, A. R. Pavan, Victor Gabriel de Faria Pastre et al. · 0 citations
Open access Jul 2026

Biallelic GTF3A mutations underline a novel human combined immunodeficiency

Compound heterozygous mutations in GTF3A, encoding transcription factor IIIA, are identified in a patient with CID presenting with profound T-cell lymphopenia, diminished thymic output, and humoral failure, establishing deficiency of GTF3A resulting in combined immunodeficiency (DoGCID) within the spectrum of ribosomop...

L. Yu, H. Li, Q. Wei et al. · 0 citations
Open access Aug 2026

Biomarker implications of gene editing in hematologic diseases: latest updates from ASH 2025

Overall, ASH 2025 suggests a shift from achieving gene editing to quantifying efficacy, durability, and safety, with standardized biomarker frameworks likely to play an increasingly important role in future clinical translation.

Ze-Zhou Zang, Wei Zheng, Rui-Rong Xu et al. · 0 citations

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