Associated Papers
Preclinical efficacy of a modified gamma-globin lentivirus gene therapy in Berkeley sickle cell anemia mice and human xenograft models.
Shrestha A et al.
Molecular Therapy. Methods & Clinical Development, 2025
A novel high-titer, bifunctional lentiviral vector for autologous hematopoietic stem cell gene therapy of sickle cell disease.
Hart KL et al.
Molecular Therapy. Methods & Clinical Development, 2024
38745893 (PubMed )
Supplement: mmc2.pdf
Pre-clinical Development of a Lentiviral Vector Expressing the Anti-sickling βAS3 Globin for Gene Therapy for Sickle Cell Disease.
Poletti V et al.
Molecular Therapy. Methods & Clinical Development, 2018
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