Stem Cell Research

Sickle Cell Disease Resources

What is Sickle Cell Disease?

Sickle Cell Disease (SCD) is a genetic disease that affects hemoglobin, a protein in red blood cells, which carries oxygen from the lungs to the rest of the body. Hemoglobin structure is determined by the genes inherited from each parent.

A person with SCD must inherit an abnormal copy of the hemoglobin gene (i.e. HbS, HbC, etc.) from both of their parents. Healthy red blood cells with normal hemoglobin are flexible and able to easily move through the smallest blood vessels in the body.

In SCD, due to abnormal hemoglobin, the red blood cells are shaped like a C, or a sickle, and are less flexible, making them prone to becoming stuck in the small vessels and causing blockages. These blockages lead to symptoms and complications.

 

Sickle vs Normal RBC

 

 

Frequently Asked Questions about Sickle Cell Disease

 UCLA Health Sickle Cell Program Website  

To establish care for Sickle Cell Disease at UCLA, you can contact the UCLA Health Sickle Cell Program at (310) 267-1654 or email: sicklecell@mednet.ucla.edu.

The program offers the following services:

  • High quality primary care with primary care champions
  • Comprehensive oversight by sickle cell providers with access to world-class infusion and transfusion support
  • World-class specialists in Pulmonary, Endocrine, Orthopedics and others to address any potential complication
  • Neuro-Psychiatric assessment
  • Adolescence to adult program to help sickle cell youth advocate for themselves
  • Access to cutting edge therapy for sickle cell led by our own researchers

Now available at the following locations: Downtown Los Angeles, Redondo Beach, Santa Clarita, Westwood

You can also view the UCLA Sickle Cell Program flyer (PDF) here: 

In 2023, the FDA approved two gene therapy treatments for sickle cell—Vertex and CRISPR Therapeutics’ Casgevy and bluebird bio’s Lyfgenia—bringing forth a new era of treatment options for people living with the condition. CIRM recognizes that more research and long-term innovation is needed to lower the costs of gene therapies, reduce the burden of the treatment on the patient, and make sickle cell therapies more accessible.

See more information about clinical trial options for Sickle Cell Disease at the NIH-sponsored page: https://curesickle.org/clinical-trials/ 

To view UCLA Health clinical trials for Sickle Cell Disease, please visit the UCLA Health Clinical Trials Page.

CureSickle.org - https://www.nhlbi.nih.gov/science/cure-sickle-cell-initiative/ 
The Cure Sickle Cell Initiative is an NHLBI-led collaborative research effort that is accelerating the development of gene therapies to cure sickle cell disease

CIRM: Championing Change for Sickle Cell Disease in California - https://www.cirm.ca.gov/sickle-cell-disease/
The California Institute for Regenerative Medicine (CIRM) is funding regenerative medicine and gene therapy research to improve the lives of Sickle Cell patients in California and the world. This page is filled with resources for patients, caregivers, investigators and study teams.

 

Sickle Cell Disease and Clinical Trials News

Lynndrick Holmes

Defeating Sickle Cell Disease by Enrolling in NIH Gene Therapy Trial

Hosted by the UCLA CTSI Community Engagement and Research Program, this webinar will delve deep into the barriers faced while navigating a gene therapy trial conducted by the National Institutes of Health to cure Mr. Lynndrick Holmes of sickle cell disease.

Click to watch this webinar on YouTube
Evie James Jr.

UCLA gene therapy trial for sickle cell disease: Evie’s story

For Evie Junior, living with sickle cell disease has been like running a marathon.

“But it’s a marathon where as you keep going, the trail gets rockier and then you lose your shoes,” the 27-year-old said.

Click to watch Evie's story on YouTube
CRISPR Trial

UCSF, Berkeley, UCLA to Launch Sickle Cell Trial Using CRISPR (2023)

UC Consortium launches first clinical trial using CRISPR to correct the gene defect that causes Sickle Cell Disease.

Click to watch video on YouTube
UCLA Still

New gene therapy for sickle cell disease has been a long time coming

But is it a complete game-changer? ‘In Los Angeles County, the life expectancy for adult sickle cell patients is the lowest among all the counties in the U.S.,’ says UCLA Health’s Dr. Gary J. Schiller.

Click here to read the UCLA full article
FDA.Gov Article

FDA Approves First Gene Therapies to Treat Patients with SCD

On December 8, 2023, the U.S. Food and Drug Administration approved two milestone treatments, Casgevy and Lyfgenia, representing the first cell-based gene therapies for the treatment of sickle cell disease (SCD) in patients 12 years and older.

Click to read the article on FDA.gov
NIH_Article

FDA approval of gene therapies for sickle cell disease

FDA approval of gene therapies for sickle cell disease: Q&A with NHLBI Director Dr. Gary Gibbons and NHLBI’s Division of Blood Diseases and Resources Director Dr. Julie Panepinto

Click to read the article on the NIH website