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Single-cell and TCR Sequencing Analysis of Peripheral Blood Mononuclear Cells from Duchenne Muscular Dystrophy Patients Before and after Dosing with AAV9 Mini-Dystrophin

Michael R. Emami,Mark A. Brimble,Alejandro Espinoza,Jane Owens,Laurence O. Whiteley,Sandra Casinghino,Thomas A. Lanz,Philip K. Farahat,Matteo Pellegrini,Courtney S. Young,Paul G. Thomas,Elizabeth M. McNally,S. Armando Villalta,Stefan A. Schattgen,Melissa J. Spencer

Molecular Therapy - Methods &amp Clinical Development（2024）

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