Ultra-rare diseases offer insights into fascinating new biology: A focus on red blood cell production and the hormone erythropoietin.

Semester: 

Summer

Offered: 

2023

Course Overview:

Each one of us has 25 trillion red blood cells, and we make 250 billion new red blood cells each day. Red blood cell production is tightly regulated by the production of erythropoietin, a hormone that is secreted by the kidneys but has its effect in the bone marrow. This nanocourse explores the history of erythropoietin, how its production is tightly regulated, rare diseases that dysregulate erythropoietin regulation, and new classes of medications that stimulate erythropoietin production. While we focus on erythropoietin, we will also think about and explore how rare mistakes of nature can lead to new insights into fundamental aspects of biology.

Course Learning Objectives:

  • Understand how erythropoietin production is regulated at the transcriptional level.
  • Think about how very rare diseases can provide new and exciting insights into biology.
  • Consider the pathway from basic science to patient in developing new drugs that regulate red blood cell production.

Enrollment:

This course is designed for up to 20 participants. Enrollment over 20 will be considered on a case-by-case basis.

Course Schedule:

SESSION 1: Introductory session on erythropoietin and rare disease states.

Monday, May 15, 12:00pm – 1:30pm, TMEC - 227

SESSION 2: Journal club & discussion – how did erythropoietin regulation result in a Nobel Prize?

Thursday, May 18, 12:00pm – 2:30pm, TMEC - 227

SESSION 3: Journal club & presentations – drug development and new research.

Monday, May 22, 12:00pm – 2:30pm, TMEC - 227

Target Audience and Prerequisites:

This course is designed for graduate students and postdoctoral fellows. Advanced undergraduate students or others are welcome if space allows.

Course Team:

Course Director:

David B. Sykes, M.D., Ph.D. (dbsykes@mgh.harvard.edu)

Assistant Professor, Center for Regenerative Medicine, Massachusetts General Hospital

Curriculum Fellow:

Emmanuel Jose Rivera Rodriguez, Ph.D. (Emmanuel_RiveraRodriguez@hms.harvard.edu)

REGISTER HERE: 

Course Registration Form