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Novel Genome Editing Therapy Improves Disease Phenotype And Survival Of A Mouse Model Of Methylmalonic Acidemia

Amy Bastille,Xiaohan Zhang,Nikhil Ramesh,Jenisha Vora,Elizabeth Mccarthy,Dylan Frank,Chih-Wei Ko,Carmen Wu,Noel Walsh,Jing Liao,Susana Gordo,Lauren Drouin,Matthias Hebben,Kyle Chiang,Nelson Chau,Shengwen Zhang

MOLECULAR THERAPY（2021）

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