About the Authors

Yimin Hua

Affiliation Cold Spring Harbor Laboratory, Cold Spring Harbor, New York, United States of America

Timothy A Vickers

Affiliation Isis Pharmaceuticals, Carlsbad, California, United States of America

Brenda F Baker

Affiliation Isis Pharmaceuticals, Carlsbad, California, United States of America

C. Frank Bennett

Affiliation Isis Pharmaceuticals, Carlsbad, California, United States of America

Adrian R Krainer

To whom correspondence should be addressed. E-mail: krainer@cshl.edu

Affiliation Cold Spring Harbor Laboratory, Cold Spring Harbor, New York, United States of America

Competing Interests

TAV, BFB, and CFB are employees of Isis Pharmaceutical Corp., the owner of the antisense oligonucleotide chemistry used in this report, and materially benefit either directly or indirectly through stock options. YH and ARK, along with their employer, Cold Spring Harbor Laboratory, could materially benefit if a therapeutic for SMA results from this work. ARK serves on the scientific advisory board of two non-profit SMA foundations.

Author Contributions

YH, TAV, BFB, CFB, and ARK conceived and designed the experiments. YH performed the experiments. YH and ARK analyzed the data. YH, TAV, BFB, CFB, and ARK contributed reagents/materials/analysis tools. YH and ARK wrote the paper.