Genetic correction
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Genetic Correction of Inherited Hemoglobin Disease
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Transcript of Genetic correction
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Genetic Correction of Inherited Hemoglobin Disease
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300 million families with Hb gene diseases
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CRISPR-CAS9
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Treatment strategy
No Hb = disease
Patient mutations
Hemoglobin gene
Cure with functional fetal Hb
CRISPR THERAPY
Fetal hemoglobin gene
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Fetal hemoglobin (HbG/ HbA)
100% fetal Hb increase after genetic modification
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Untreated cells
Genetically modifiedcells
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Treatment benefiting millions by 2035
2015 2035
• Scientific proof of principle
• 2 innovation disclosures• 2 research hospitals• World class mentors
• Investors• International biotech• Clinical trials for safety &
efficacy
• Ethics• Politics
2025