1. A-44G transition in SMN2 intron 6 protects patients with spinal muscular atrophy. (28th April 2017) Authors: Wu, Xingxing; Wang, Shu-Huei; Sun, Junjie; Krainer, Adrian R; Hua, Yimin; Prior, Thomas W Journal: Human molecular genetics Issue: Volume 26:Number 14(2017:Jul. 15) Page Start: 2768 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
2. Antisense oligonucleotides correct the familial dysautonomia splicing defect in IKBKAP transgenic mice. Issue 10 (17th April 2018) Authors: Sinha, Rahul; Kim, Young Jin; Nomakuchi, Tomoki; Sahashi, Kentaro; Hua, Yimin; Rigo, Frank; Bennett, C Frank; Krainer, Adrian R Journal: Nucleic acids research Issue: Volume 46:Issue 10(2018) Page Start: 4833 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
3. Comparison of the efficacy of MOE and PMO modifications of systemic antisense oligonucleotides in a severe SMA mouse model. Issue 6 (27th February 2020) Authors: Sheng, Lei; Rigo, Frank; Bennett, C Frank; Krainer, Adrian R; Hua, Yimin Journal: Nucleic acids research Issue: Volume 48:Issue 6(2020) Page Start: 2853 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
4. Disruption of splicing-regulatory elements using CRISPR/Cas9 to rescue spinal muscular atrophy in human iPSCs and mice. Issue 1 (3rd September 2019) Authors: Li, Jin-Jing; Lin, Xiang; Tang, Cheng; Lu, Ying-Qian; Hu, Xinde; Zuo, Erwei; Li, He; Ying, Wenqin; Sun, Yidi; Lai, Lu-Lu; Chen, Hai-Zhu; Guo, Xin-Xin; Zhang, Qi-Jie; Wu, Shuang; Zhou, Changyang; Shen, Xiaowen; Wang, Qifang; Lin, Min-Ting; Ma, Li-Xiang; Wang, Ning Journal: National science review Issue: Volume 7:Issue 1(2020) Page Start: 92 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
5. Downregulation of Survivin contributes to cell-cycle arrest during postnatal cardiac development in a severe spinal muscular atrophy mouse model. (6th December 2017) Authors: Sheng, Lei; Wan, Bo; Feng, Pengchao; Sun, Junjie; Rigo, Frank; Bennett, C Frank; Akerman, Martin; Krainer, Adrian R; Hua, Yimin Journal: Human molecular genetics Issue: Volume 27:Number 3(2018:Feb. 01) Page Start: 486 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
6. Hybridization-mediated off-target effects of splice-switching antisense oligonucleotides. Issue 2 (5th December 2019) Authors: Scharner, Juergen; Ma, Wai Kit; Zhang, Qian; Lin, Kuan-Ting; Rigo, Frank; Bennett, C Frank; Krainer, Adrian R Journal: Nucleic acids research Issue: Volume 48:Issue 2(2020) Page Start: 802 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗