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Rachel Ciotti
Rachel Ciotti
Genetic enhancement
Clinical trial
Disease
Viral vector
Oncology
2
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1
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0
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The GuardOne clinical trial: A first-in-human, open-label, multinational phase 1/2 study of AVR-RD-02 ex vivo lentiviral vector, autologous gene therapy for Gaucher disease
2021
Molecular Genetics and Metabolism
Leslie Jacobsen
Julie A. Kerner
Rachel Ciotti
Azadeh Golipour
Josie Yang
Richard Pfeifer
Aneal Khan
Christopher E. Mason
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Analysis of genetically engineered stem cell product and follow up of gene therapy patients through high-throughput single cell technologies
2021
Molecular Genetics and Metabolism
Cristina Baricordi
Mariana Loperfido
Aimin Yan
Hareesh Chamarthi
Luigi Barbarossa
Azadeh Golipour
Rachel Ciotti
Aneal Khan
Leslie K. Jacobsen
Christopher E. Mason
Luca Biasco
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