What's YOur Status? 

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what's happening now ?

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Let's talk about it- the cure for sickle cell disease. 
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Are you interested in curative treatment for sickle cell disease but you have questions? Want to know the difference between gene therapy with CRISPR-Cas9 technique and gene therapy utilizing a lentiviral vector aka (gene delivery vehicle)?
Check out the recording with Dr. Frangoul because we've got answers.
Dr. Frangoul, is a board-certified pediatric hematologist/oncologist and the medical director of pediatric hematology/oncology for the Sarah Cannon Pediatric Transplant and Cellular Therapy Program at TriStar Centennial. He also serves as an investigator for Sarah Cannon Research Institute, and he serves as the lead investigator for the CRISPR-Cas 9 gene editing trial for sickle cell disease. 

 Replay:
​https://drive.google.com/file/d/1mnEzTCgeACD1xOC5qrFCtJ4H-VIJGl1I/view?usp=drive_link
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​[email protected]
Haydar Frangoul, MD, MS
Program Director
Sarah Cannon Pediatric Hematology/Oncology &
Cellular Therapy @TriStar Centennial
330 23rd Ave North, Suite 450
Nashville, TN 37203
O Office 615-342-7339 F Fax 615-340-4099

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Gene Therapy in Review

Lyfgenia and Casgevy are both one-time gene therapies used to treat sickle cell disease in individuals 12 years of age or older and a history of vaso-occlusive events.

 Learn more. 

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how CASGEVY™ (exagamglogene autotemcel) works

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​BTSSCYCLE Community Impact 

​From Chasity S. and Shelby:
​I met Dr. Scott back in January 2017 at a Christmas party that Dr. Debaun and the Vanderbilt team have yearly for the SCD youth . It was actually Jeannie (Vanderbilt Pediatric Sickle Cell Case Manager), that introduced me to her. It wasn’t until August 2018, that I asked if she would come speak with the educators at my daughters school about SCD. My daughter (Shelby) was 5yrs old attending a private school (EZELL Harding Christian School). At the time Shelby battled in/out hospitalization stays and needed frequent restroom breaks.
I requested for 504 plan to accommodate her needs at the school. Unfortunately, at that time her teacher wasn’t supportive of a 504 plan as she did not understand Shelby’s medical condition. As a parent my back was against the wall because I had no support in the school. 
I reached out to Dr. Scott/BTSSCYCLE by Facebook and asked if she would share her knowledge about SCD to the administrative staff at Shelby's school. I was initially hesitant, because I never thought I would be the one needing help educating teachers about SCD. I waited patiently as I wasn’t sure if she would provide that type of service of going into a private school. 
She responded right away and I was totally blown away with excitement! I immediately notified the principal at the school and she made it a priority for the teachers to attend the meeting about SCD. 
After the meeting staff were so humbled by the wealth of information provided to them. I would receive a call a few days later to setup an 504 plan for Shelby. I have  also received resources in the community financially and medically from Breaking The SSickle Cell Cycle Foundation.
I will say before working with Dr. Scott/Breaking The SSickle Cell Cycle Foundation., I felt hopeless and powerless within the private school sector. Having  Dr. Scott and Breaking The SSickle Cell Cycle Foundation support me, I feel confident knowing support is there.
Chasity S. 

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WHAT IS ​Sickle Cell Disease (SCD) ? 

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SICKLE CELL FACTS.

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What Causes
Sickle Cell Disease

GENETICS. 
SCD is a genetic condition that is present at birth. It is inherited when a child receives two sickle cell genes—one from each parent.

Symptoms and Treatment 


People with sickle cell disease can start to have signs of the disease during the first year of life, usually around 5 months of age. Pain is the hallmark sign of sickle cell disease.
​Symptoms and complications of SCD are different for each person and can range from mild to severe. 

Cure
Is There A Cure

Yes! A blood or marrow transplant  is the only known cure for SCD. Bone marrow is a soft, fatty tissue inside the center of the bones where blood cells are made. A bone marrow or stem cell transplant is a procedure that takes healthy cells that form blood from one person—the donor—and puts them into someone whose bone marrow is not working properly.

Sickle Cell Trait
Sickle cell trait is an inherited blood disorder that affects 1 million to 3 million Americans and 8 to 10 percent of African Americans. Sickle cell trait can also affect Hispanics, South Asians, Caucasians from southern Europe, and people from Middle Eastern countries. More than 100 million people worldwide have sickle cell trait.

Genetic Blood Disorder
Unlike sickle cell disease, a serious illness in which patients have two genes that cause the production of abnormal hemoglobin (the substance in red blood cells that helps carry oxygen), individuals with sickle cell trait carry only one defective gene, have normal functioning hemoglobin and typically live normal lives.

Newborn Testing
All newborns in the United States are now tested for sickle cell disease and sickle cell trait. Sickle cell disease can be identified before birth by testing a sample of amniotic fluid or tissue from the placenta. People who carry the sickle cell gene can seek genetic counseling before pregnancy to discuss options.

​DIAGNOSIS.

SCT is diagnosed with a simple blood test. People at risk of having SCT can talk with a doctor or health clinic about getting this test.

COMPLICATIONS.

Most people with SCT do not have any symptoms of SCD, although—in rare cases—people with SCT might experience complications of SCD, such as pain crises.