SICKLE CELL STUDIES
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PRESENCE Study

Living with sickle cell can mean dealing with pain that comes out of nowhere.

Locations

United States

Age

>

16 to 30 years old

Years

Genotypes

All types of SCD

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Transplantation of Clustered Regularly Interspaced Short Palindromic Repeats Modified Hematopoietic Progenitor Stem Cells (CRISPR_SCD001) in Patients With Severe Sickle Cell Disease

This study seeks to understand the safety of a gene editing therapy called CRISPR_SCD001 and if this treatment can help people with severe sickle cell disease get better.

Locations

2 United States of America sites

Age

>

12-35 years

Years

Genotypes

SCD type SS

Phase

1/2

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