News & Events

Picture of Robyn Loves with the following text: Robyn Loves, PhD University of California, Los Angeles, Department of Microbiology, Immunology & Molecular Genetics
Events November 3, 2026

Lentiviral Gene Therapy for Alpha Thalassemia: Toward Clinical Application
Picture of Tippi MacKenzie with the following text: Tippi MacKenzie, MD University of California, San Francisco Department of Surgery
Events

In Utero Hematopoietic Stem Cell Transplantation for the Treatment of Fetuses with Alpha Thalassemia Major

May 20, 2026
Photo of man with short brown against a background of abstract blood cells Khaled Musallam
Events

Current Understanding of Alpha Thalassemia Burden: Evidence and Gaps

January 29, 2026
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News Research

Antisense oligonucleotides delivered into amniotic fluid improve spinal muscular atrophy in mice

May 20, 2025
Young woman with long brown hair smiles against a background of abstract blood cells Evangelia Vlachodimitropoulou
Events

In Utero Transfusions Beyond 35 Weeks Gestation in Alpha Thalassemia Major

Evangelia Vlachodimitropoulou, MBBS, PhD Thursday, June 12, 2025, 8:00-9:00 am (San Francisco) In this session: – The International Alpha Thalassemia […]

June 12, 2025
Center Fellow Akos Herzeg, MD, and colleagues have conducted a scoping review of current literature to better understand the causes and the genetic basis of primary hydrocephalus. Among the over 160 genes linked to this condition, single-gene mutations in L1CAM, TRIM71, MPDZ, and CCDC88C have emerged to play a critical role in the development of primary hydrocephalus. Some may represent promising targets for prenatal somatic cell gene therapy, potentially preventing pathogenic phenotypes, improving neurodevelopmental outcomes, and reducing the need for pre- or postnatal surgical approaches. Considering the emerging nature of prenatal molecular therapies and the absence of established guidelines, this review provides a comprehensive overview of current knowledge, encouraging the clinical and scientific community to reassess and refine practices for optimal assessment and management.
News

The Conundrum of Mechanics Versus Genetics in Congenital Hydrocephalus and Its Implications for Fetal Therapy Approaches: A Scoping Review

Center Fellow Akos Herzeg, MD, and colleagues have conducted a scoping review of current literature to better understand the causes […]

September 10, 2024
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Events

Alpha Thalassemia Major Beyond the Neonatal Period: From a Pond of Knowledge to an Ocean of Unknowns

Ali Amid, MD Thursday, September 26, 2024, 8:00-9:00 am (San Francisco) In this session: Alpha thalassemia major was once considered […]

September 26, 2024