Lecture 20: Thalassemia

Sujit Sheth MD

Presentation Date:

February 20, 2025

> Quantitative Hemoglobinopathies
(disorders of ineffective erythropoiesis).

> Globin gene mutations that result in decreased production of globin chains
Thalassemias (Alpha, Beta, Gamma, Delta).

Chair, Pediatric Hematology/Oncology,
Weill Cornell Medicine
shethsu@med.cornell.edu

> Consultant
Novonordisk
Celgene / Bristol Myers Squibb
Agios
> Steering Committeecommittee
CRISPR/ Vertex CTX001
Will discuss therapeutics not yet FDA approved – results from clinical trials

Left: Globin Structure of Hemoglobin. https://upload.wikimedia.org/wikipedia/commons/2/23/Oxy-Hemoglobin.jpg

Starting in 2025, lectures are eligible for Continuing Education Credit.
Link for CE:

Slide Deck