Author
Harvard University
Listed by name: these papers carry no ORCID for this author, so different people with the same name may be mixed together.
5 papers
Recent·Most cited
Christiano R. R. Alves, Sabyasachi Das, Vijai Krishnan…·Patricia Leonor Musolino
This paper develops a customized CRISPR-Cas9 base editor to correct the ACTA2 R179H mutation in a mouse model of MSMDS, improving survival and rescuing vascular and neurological phenotypes.
Miguel C. Santoscoy, Paula Espinoza, Killian S. Hanlon…·Casey A. Maguire
The paper identifies an AAV capsid, MC5, that efficiently and specifically transduces microglia via direct and intracerebroventricular injection in mice.
Killian S. Hanlon, Ming Liang Cheng, Roberto Montoro Ferrer…·Casey A. Maguire
The paper identifies superior AAV capsids for targeted spinal cord gene therapy in non-human primates through in vivo selection and evaluates their biodistribution and expression.
Christiano R. R. Alves, Leillani L. Ha, Rebecca Yaworski…·Benjamin P. Kleinstiver
This paper describes the optimization of base editing to correct the SMN2 exon 7 mutation in SMA, achieving high editing efficiency and increased SMN protein levels in vitro and in vivo.