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Last updated about 15 hours ago
Long-term outcomes of lentiviral gene therapy for the β-hemoglobinopathies: the HGB-205 trial
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Antisense oligonucleotide silencing of FUS expression as a therapeutic approach in amyotrophic lateral sclerosis
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Long-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott–Aldrich syndrome
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