Patient resources for sickle cell disease gene therapy

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Understanding sickle cell disease
Understanding gene therapy
FDA-approved gene therapies for sickle cell disease
Families and caregivers
Mental health
Clinical trials
Other helpful resources

Understanding sickle cell disease

What is Sickle Cell Disease? National Institutes of Health (NIH)

About Sickle Cell Disease Centers for Disease Control and Prevention

Sickle Cell Disease American Society of Hematology (ASH)

What is Sickle Cell Anemia? (video) American Society of Hematology (ASH)

Understanding Sickle Cell Disease (handout) American Society of Hematology (ASH)

Understanding sickle cell disease

What is Sickle Cell Disease? National Institutes of Health (NIH)
About Sickle Cell Disease   Centers for Disease Control and Prevention
Sickle Cell Disease American Society of Hematology (ASH)
What is Sickle Cell Anemia? (video)   American Society of Hematology (ASH)
Understanding Sickle Cell Disease (handout) American Society of Hematology (ASH)

Understanding gene therapy

Gene Therapy and Sickle Cell Disease (video) American Society of Gene & Cell Therapy (ASGCT)

Gene Therapy: What you need to know Sickle Cell Disease Association of America (SCDAA)

What’s the Difference Between Gene Therapy, Cell Therapy and Gene Editing? (video) American Society of Gene & Cell Therapy (ASGCT)

Gene Therapy Basics American Society of Gene & Cell Therapy (ASGCT)

Gene Therapy Basics (video) American Society of Gene & Cell Therapy (ASGCT)

What is Gene Therapy? U.S. Food and Drug Administration (FDA)

Gene Therapies for Sickle Cell Disease Cure Sickle Cell

Gene Editing American Society of Gene & Cell Therapy (ASGCT)

Gene Therapy Conditioning Cure Sickle Cell

Vectors 101 American Society of Gene & Cell Therapy (ASGCT)

Sickle Cell Disease Gene Therapy Clinical Trials ClinicalTrials.gov

Understanding gene therapy

Gene Therapy and Sickle Cell Disease (video) American Society of Gene & Cell Therapy (ASGCT)
Gene Therapy: What you need to know Sickle Cell Disease Association of America (SCDAA)
What’s the Difference Between Gene Therapy, Cell Therapy and Gene Editing? (video) American Society of Gene & Cell Therapy (ASGCT)
Gene Therapy Basics American Society of Gene & Cell Therapy (ASGCT)
Gene Therapy Basics (video) American Society of Gene & Cell Therapy (ASGCT)
What is Gene Therapy? U.S. Food and Drug Administration (FDA)
Gene Therapies for Sickle Cell Disease Cure Sickle Cell
Gene Editing American Society of Gene & Cell Therapy (ASGCT)
Gene Therapy Conditioning   Cure Sickle Cell
Vectors 101 American Society of Gene & Cell Therapy (ASGCT)
Sickle Cell Disease Gene Therapy Clinical Trials ClinicalTrials.gov

FDA-approved gene therapies for sickle cell disease

Exagamglogene autotemcel

Exagamglogene autotemcel MedlinePlus, National Library of Medicine
CASGEVY – exagamglogene autotemcel injection, suspension DailyMed, National Library of Medicine
FDA Approves First Gene Therapies to Treat Patients with Sickle Cell Disease
Casgevy FDA

Lovotibeglogene autotemcel

Lovotibeglogene autotemcel Injection MedlinePlus, National Library of Medicine
LYFGENIA – lovotibeglogene autotemcel suspension DaliyMed, National Library of Medicine
FDA Approves First Gene Therapies to Treat Patients with Sickle Cell Disease
Lyfgenia FDA

FDA-approved gene therapies for sickle cell disease
Exagamglogene autotemcel
Exagamglogene autotemcel MedlinePlus, National Library of Medicine
CASGEVY – exagamglogene autotemcel injection, suspension DailyMed, National Library of Medicine
FDA Approves First Gene Therapies to Treat Patients with Sickle Cell Disease
Casgevy FDA

Lovotibeglogene autotemcel
Lovotibeglogene autotemcel Injection MedlinePlus, National Library of Medicine
LYFGENIA – lovotibeglogene autotemcel suspension DaliyMed, National Library of Medicine
FDA Approves First Gene Therapies to Treat Patients with Sickle Cell Disease
Lyfgenia FDA

Families and caregivers

Sickle Cell Disease: Information for Parents HealthChildren.org

Information for Families about Sickle Cell Disease Centers for Disease Control and Prevention (CDC)

Disability & Health Information for Family Caregivers Centers for Disease Control and Prevention (CDC)

Caregivers of Children with Rare and/or Serious Illnesses National Alliance for Caregiving

Caregiver Burnout Cleveland Clinic

Families and caregivers

Sickle Cell Disease: Information for Parents HealthChildren.org
Information for Families about Sickle Cell Disease Centers for Disease Control and Prevention (CDC)
Disability & Health Information for Family Caregivers Centers for Disease Control and Prevention (CDC)
Caregivers of Children with Rare and/or Serious Illnesses National Alliance for Caregiving
Caregiver Burnout Cleveland Clinic

Mental health

CDC Mental Health Tools and Resources Centers for Disease Control and Prevention (CDC)

Mental Health and Wellness Sickle Cell Disease Association of America (SCDAA)

I’m So Stressed Out! (fact sheet) National Institute of Mental Health (NIMH)

Mental health

CDC Mental Health Tools and Resources Centers for Disease Control and Prevention (CDC)
Mental Health and Wellness Sickle Cell Disease Association of America (SCDAA)
I’m So Stressed Out! (fact sheet) National Institute of Mental Health (NIMH)

Clinical trials

Questions to Ask About Volunteering for a Research Study U.S. Department of Health and Human Services (HHS)

Human Research Volunteer Informational Videos U.S. Department of Health and Human Services (HHS)

NIH Clinical Research Trials and You – The Basics National Institutes of Health (NIH)

Learn About Studies ClinicalTrials.gov

Clinical trials

Questions to Ask About Volunteering for a Research Study U.S. Department of Health and Human Services (HHS)
Human Research Volunteer Informational Videos U.S. Department of Health and Human Services (HHS)
NIH Clinical Research Trials and You – The Basics National Institutes of Health (NIH)
Learn About Studies ClinicalTrials.gov

Other helpful resources

Cure Sickle Cell Resource Page Cure Sickle Cell

Talking With Your Doctor or Health Care Provider National Institutes of Health (NIH)

Social Security Administration and SCD Disability Guide

National Alliance for Sickle Cell Centers

Other helpful resources

Cure Sickle Cell Resource Page Cure Sickle Cell
Talking With Your Doctor or Health Care Provider National Institutes of Health (NIH)
Social Security Administration and SCD Disability Guide
National Alliance for Sickle Cell Centers

a family with a young girl high-fiving her doctor

Sickle cell disease gene therapy education project

The Democratizing Education for Sickle Cell Disease Gene Therapy project developed patient-focused educational materials for the sickle cell community.

Understanding the blood cell

Learn about the different types of hemoglobin.

Understanding gene therapy approaches

A variety of approaches target the blood stem cells to relieve or reduce your symptoms of sickle cell disease.

Navigating sickle cell disease gene therapy

Gene therapy for sickle cell disease is available to you through FDA-approved therapies and through clinical trials.

Your mental health and gene therapy

Your mental and physical health are deeply connected. Mental health is important for your overall health and quality of life.

Sickle cell disease gene therapy FAQ

Discuss these questions with your doctor or health care provider.

a group of four healthcare workers seated at a table having a conversation

Resources for healthcare providers

Sickle cell disease resources for healthcare providers

The Democratizing Education Project welcomes your feedback about the sickle cell disease gene therapy resources. Please email your comments or questions to DemocratizingEd@mail.nih.gov.

These educational materials are for informational purposes only. They are meant to promote your general understanding of gene therapy for sickle cell disease. We encourage you to use these educational materials to talk with your healthcare provider or a clinical trial team.

Last updated: December 12, 2024