Author

Anas M. Alazami

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Open access Sep 2026

Hematopoietic stem cell transplantation for DOCK8 deficiency: durable immune reconstitution and long-term survival in 47 patients

Background DOCK8 deficiency is a severe autosomal recessive combined immunodeficiency characterized by recurrent infections, severe atopy, immune dysregulation, and an increased risk of malignancy. Allogeneic hematopoietic stem cell transplantation (HSCT) remains the only curative treatment. We aimed to evaluate the lo...

Bashayr Alanazi, R. Mohammed, A. Al-Ahmari et al. · Frontiers in Immunology · 0 citations

Open access Jul 2026

A TLS11a-decorated ionizable lipid nanoparticle platform and a multilevel-validated CRISPR LDLR-knockout HepG2 model for hepatocyte-preferential mRNA delivery

A modular aptamer-guided LNP platform to enable hepatocyte-selective genome editing through an LDLR-independent route is designed and validated and a validated LDLR-null hepatocyte model is validated for studying LDLR-dependent biology and disease.

Irfan Hussain, Naji Kholaif, Raghad Alsultan et al. · bioRxiv · 0 citations

Case report Open access Aug 2026

A Novel SLC25A4 Variant Causing Mitochondrial Dysfunction, Myopathy and Cardiomyopathy: A Functional and Molecular Characterization

The molecular and functional spectrum of SLC25A4-associated disease is expanded and may inform clinical practice, including genetic interventions such as preimplantation genetic diagnosis, premarital genetic screening, targeted genetic counseling, and cascade testing of at-risk family members.

Mazhor Aldosary, Hanan Alqudairy, Nourah Alshalan et al. · International Journal of Mol... · 0 citations