Dakai Mu
Harvard University
12 Papers
50 Citations
Dakai Mu is an academic researcher from Harvard University. The author has contributed to research in topics: Gene delivery & Biology. The author has an hindex of 10, co-authored 12 publications.
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Papers
Rescue of Hearing by Gene Delivery to Inner-Ear Hair Cells Using Exosome-Associated AAV
Bence György,Cyrille Sage,Artur A. Indzhykulian,Deborah I. Scheffer,Alain Brisson,Sisareuth Tan,Xudong Wu,Adrienn Volak,Dakai Mu,Panos I. Tamvakologos,Yaqiao Li,Zachary Fitzpatrick,Maria Ericsson,Xandra O. Breakefield,David P. Corey,Casey A. Maguire +15 more
TL;DR: Exo-AAV is a powerful gene delivery system for hair cell research and may be useful for gene therapy for deafness and shows no toxicity in vivo, as assayed by tests of auditory and vestibular function.
Heparin affinity purification of extracellular vesicles.
Leonora Balaj,Nadia A. Atai,Weilin Chen,Dakai Mu,Bakhos A. Tannous,Xandra O. Breakefield,Johan Skog,Casey A. Maguire +7 more
TL;DR: UF/heparin-purified EVs from cell culture displayed the EV marker Alix, contained a diverse RNA profile, had lower levels of protein contamination, and were functional at binding to and uptake into cells, supporting a direct EV- heparin interaction.
CRISPR/Cas9 Mediated Disruption of the Swedish APP Allele as a Therapeutic Approach for Early-Onset Alzheimer's Disease.
Bence György,Camilla Lööv,Mikołaj Piotr Zaborowski,Mikołaj Piotr Zaborowski,Shuko Takeda,Benjamin P. Kleinstiver,Caitlin Commins,Ksenia V. Kastanenka,Dakai Mu,Adrienn Volak,Vilmantas Giedraitis,Lars Lannfelt,Casey A. Maguire,J. Keith Joung,Bradley T. Hyman,Xandra O. Breakefield,Martin Ingelsson,Martin Ingelsson +17 more
TL;DR: Proof-of-concept data is presented that CRISPR/Cas9 can selectively disrupt the APPSW allele both ex vivo and in vivo—and thereby decrease pathogenic Aβ.
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Naturally enveloped AAV vectors for shielding neutralizing antibodies and robust gene delivery in vivo.
TL;DR: It is demonstrated that in mice, AAV vectors associated with extracellular vesicles (EVs) can evade human anti-AAV neutralizing antibodies and it is shown that expressing a brain targeting peptide on the EV surface allowed significant enhancement of transduction compared to untargeted ev- AAV.
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Exosome-associated AAV vector as a robust and convenient neuroscience tool.
Eloise Hudry,Courtney Martin,Sheetal Gandhi,Bence György,Deborah I. Scheffer,Dakai Mu,Steven F. Merkel,Federico Mingozzi,Zachary Fitzpatrick,Hemi Dimant,Marissa Masek,Tim Ragan,Sisareuth Tan,Alain Brisson,Servio H. Ramirez,Bradley T. Hyman,Casey A. Maguire +16 more
TL;DR: It is demonstrated that simple pelleting of exo-AAV from media via ultracentrifugation results in high-titer vector preparations capable of efficient transduction of central nervous system (CNS) cells after systemic injection in mice, and the ability of exosomes to evade neutralizing antibodies while still transducing CNS after peripheral delivery is clinically relevant.
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