PUBLICATIONS

Cancer Discovery

for Wan et al. (2023)

Cell

for Marasco et al. (2022)

Cell

for Ruskin, Krainer, Maniatis, and Green (1984)

2024

Specificity, Synergy, and Mechanisms of Splice-Modifying Drugs

Ishigami Y, Wong MS, Martí-Gómez C, Ayaz A, Kooshkbaghi M, Hanson SM, McCandlish DM, Krainer AR, Kinney JB

Nature Communications

[CSHL Press Release]

Opportunities and Challenges for Innovative and Equitable Healthcare

Ecker DJ, Aiello CD, Arron JR, Bennett CF, Bernard A, Breakefield XO, Broderick TJ, Callier SL, Canton B, Chen JS, Fishburn CS, Garrett B, Hecht SM, Janowitz T, Kliegman M, Krainer AR, Louis CU, Lowe C, Sehgal A, Tozan Y, Tracey KJ, Urnov F, Wattendorf D, Williams TW, Zhao X, Hayden MR.

Nature Reviews Drug Discovery

Mechanistically Based Blood Proteomic Markers in the TGF-β Pathway Stratify Risk of Hepatocellular Cancer in Patients with Cirrhosis

Xiang X, Bhowmick K, Shetty K, Ohshiro K, Yang X, Wong LL, Yu H, Latham PS, Satapathy SK, Brennan C, Dima RJ, Chambwe N, Sharifova G, Cacaj F, John S, Crawford JM, Huang H, Dasarathy S, Krainer AR, He AR, Amdur RL, Mishra L

Genes Cancer

2023

Splicing Factor SRSF1 Promotes Pancreatitis and KRASG12D-Mediated Pancreatic Cancer

Wan L, Lin KT, Rahman MA, Ishigami Y, Wang Z, Jensen MA, Wilkinson JE, Park Y, Tuveson DA, Krainer AR

Cancer Discovery

[CSHL Press Release]

Antisense Oligonucleotide Therapy for H3.3K27M Diffuse Midline Glioma

Zhang Q, Yang L, Liu YH, Wilkinson JE, Krainer AR

Science Translational Medicine

[CSHL Press Release]

Preclinical Screening of Splice-Switching Antisense Oligonucleotides in PDAC Organoids [preprint]

Wan L, Kral AJ, Voss D, Krainer AR

bioRxiv

RNA Therapeutics

Hastings M, Krainer AR

RNA

RBFOX2 Modulates a Metastatic Signature of Alternative Splicing in Pancreatic Cancer

Jbara A, Lin, KT, Stossel C, Siegfried Z, Shqerat H, Amar-Schwartz A, Elyada E, Mogilevsky M, Raitses-Gurevich M, Johnson JL, Yaron TM, Ovadia O, Jang GH, Danan-Gotthold M, Cantley LC, Levanon EY, Gallinger S, Krainer AR, Golan T, Karni R

Nature

Antisense Oligonucleotide Therapeutics for Cystic Fibrosis: Recent Developments and Perspectives

Kim YJ, Krainer AR

Molecules and Cells

2022

Specificity, Synergy, and Mechanisms of Splice-Modifying Drugs [preprint]

Ishigami Y, Wong MS, Martí-Gómez C, Ayaz A, Kooshkbaghi M, Hanson S, McCandlish DM, Krainer AR, Kinney JB

bioRxiv

Higher-Order Epistasis and Phenotypic Prediction

Zhou J, Wong MS, Chen WC, Krainer AR, Kinney JB, McCandlish DM

Proceedings of the National Academy of Sciences of the United States of America

Counteracting Chromatin Effects of a Splicing-Correcting Antisense Oligonucleotide Improves Its Therapeutic Efficacy in Spinal Muscular Atrophy

Marasco LE, Dujardin G, Sousa-Luís R, Liu YH, Stigliano JN, Nomakuchi T, Proudfoot NJ, Krainer AR, Kornblihtt AR

Cell

[CSHL Press Release]

Gene-Specific Nonsense-Mediated mRNA Decay Targeting for Cystic Fibrosis Therapy

Kim YJ, Nomakuchi T, Papaleonidopoulou F, Yang L, Zhang Q, Krainer AR

Nature Communications

[CSHL Press Release]

ASO-Based PKM Splice-Switching Therapy Inhibits Hepatocellular Carcinoma Growth

Ma WK, Voss DM, Scharner J, Costa ASH, Lin KT, Jeon HY, Wilkinson JE, Jackson M, Rigo F, Bennett CF, Krainer AR

Cancer Research

[CSHL Press Release]

Systematic Characterization of Short Intronic Splicing-Regulatory Elements in SMN2 Pre-mRNA

Gao Y, Lin KT, Jiang T, Yang Y, Rahman MA, Gong S, Bai J, Wang L, Sun J, Sheng L, Krainer AR, Hua Y

Nucleic Acids Research

Exon-Skipping Antisense Oligonucleotides for Cystic Fibrosis Therapy

Kim YJ, Sivetz N, Layne J, Voss DM, Yang L, Zhang Q, Krainer AR

Proceedings of the National Academy of Sciences of the United States of America

[CSHL Press Release]

2021

Loss of KRASG12D Feedback Regulation Involving Splicing Factor SRSF1 Accelerates Pancreatic Cancer [preprint]

Wan L, Lin K, Rahman M, Wang Z, Jensen MA, Park Y, Tuveson DA, Krainer AR

bioRxiv

Towards a Combined Therapy for Spinal Muscular Atrophy Based on Opposing Effects of an Antisense Oligonucleotide on Chromatin and Splicing [preprint]

Marasco LE, Dujardin G, Sousa-Luís R, Liu Y, Stigliano JN, Nomkuchi T, Proudfoot NJ, Krainer AR, Kornblihtt AR

bioRxiv

Evidence Generation and Reproducibility in Cell and Gene Therapy Research: A Call to Action

Abou-El-Enein M, Angelis A, Appelbaum FR, Andrews NC, Bates SE, Bierman AS, Brenner MK, Cavazzana M, Caligiuri MA, Clevers H, Cooke E, Daley GQ, Dzau VJ, Ellis LM, Fineberg HV, Goldstein LSB, Gottschalk S, Hamburg MA, Ingber DE, Kohn DB, Krainer AR, Maus MV, Marks P, Mummery CL, Pettigrew RI, Rutter JL, Teichmann SA, Terzic A, Urnov FD, Williams DA, Wolchok JD, Lawler M, Turtle CJ, Bauer G, Ioannidis JPA

Molecular Therapy Methods & Clinical Developmet

Therapeutic Manipulation of IKBKAP Mis-Splicing with a Small Molecule to Cure Familial Dysautonomia

Ajiro M, Awaya T, Kim YJ, Iida K, Denawa M, Tanaka N, Kurosawa R, Matsushima S, Shibata S, Sakamoto T, Studer L, Krainer AR, Hagiwara M

Nature Communications

Gene-Specific Nonsense-Mediated mRNA Decay Targeting for Cystic Fibrosis Therapy [preprint]

Kim Y, Nomakuchi T, Papaleonidopoulou F, Krainer AR

bioRxiv

Identification of SRSF10 as a Regulator of SMN2 ISS-N1

Frederiksen SB, Holm LL, Larsen MR, Doktor TK, Andersen HS, Hastings ML, Hua Y, Krainer AR, Andresen BS.

Human Mutations

Empirical Variance Component Regression for Sequence-Function Relationships [preprint]

Zhou J, Wong MS, Chen W, Krainer AR, Kinney JB, McCandlish DM

bioRxiv

2020

SRSF1 Mediates Cytokine-Induced Impaired Imatinib Sensitivity in Chronic Myeloid Leukemia

Sinnakannu JR, Lee KL, Cheng S, Li J, Yu M, Tan SP, Ong CCH, Li H, Than H, Anczuków-Camarda O, Krainer AR, Roca X, Rozen SG, Iqbal J, Yang H, Chuah C, Ong ST

Leukemia

Comparison of the Efficacy of MOE and PMO Modifications of Systemic Antisense Oligonucleotides in a Severe SMA Mouse Model

Sheng L, Rigo F, Bennett CF, Krainer AR, Hua Y

Nucleic Acids Research

Recurrent SRSF2 Mutations in MDS Affect Both Splicing and NMD.

Rahman MA, Lin KT, Bradley RK, Abdel-Wahab O, Krainer AR

Genes & Development

[CSHL Press Release] [CSHL Press Video]

Hybridization-Mediated Off-Target Effects of Splice-Switching Antisense Oligonucleotides

Scharner J, Ma WK, Zhang Q, Lin KT, Rigo F, Bennett CF, Krainer AR

Nucleic Acids Research

SnapShot: Splicing Alterations in Cancer.

Rahman MA, Krainer AR, Abdel-Wahab O

Cell

Disruption of Splicing-Regulatory Elements Using CRISPR/Cas9 to Rescue Spinal Muscular Atrophy in Human iPSCs and Mice

Li JJ, Lin X, Tang C, Lu YQ, Hu X, Zuo E, Li H, Ying W, Sun Y, Lai LL, Chen HZ, Guo XX, Zhang QJ, Wu S, Zhou C, Shen X, Wang Q, Lin MT, Ma LX, Wang N, Krainer AR, Shi L, Yang H, Chen WJ

National Science Review

2019

PSI-Sigma: A Comprehensive Splicing-Detection Method for Short-Read and Long-Read RNA-Seq Analysis

Lin KT, Krainer AR

Bioinformatics

Differential Functions of Splicing Factors in Mammary Transformation and Breast Cancer Metastasis

Park S, Brugiolo M, Akerman M, Das S, Urbanski L, Geier A, Kesarwani AK, Fan M, Leclair N, Lin KT, Hu L, Hua I, George J, Muthuswamy SK, Krainer AR, Anczuków O

Cell Reports

Coordinated Alterations in RNA Splicing and Epigenetic Regulation Drive Leukaemogenesis

Yoshimi A, Lin KT, Wiseman DH, Rahman MA, Pastore A, Wang B, Lee SC, Micol JB, Zhang XJ, de Botton S, Penard-Lacronique V, Stein EM, Cho H, Miles RE, Inoue D, Albrecht TR, Somervaille TCP, Batta K, Amaral F, Simeoni F, Wilks DP, Cargo C, Intlekofer AM, Levine RL, Dvinge H, Bradley RK, Wagner EJ, Krainer AR, Abdel-Wahab O

Nature

[CSHL Press Release]

Antisense Oligonucleotide Therapies for Neurodegenerative Diseases

Bennett CF, Krainer AR, Cleveland DW

Annual Review of Neuroscience

Modeling RNA-Binding Protein Specificity In Vivo by Precisely Registering Protein-RNA Crosslink Sites

Feng H, Bao S, Rahman MA, Weyn-Vanhentenryck SM, Khan A, Wong J, Shah A, Flynn ED, Krainer AR, Zhang C

Molecular Cell

Delivery of GalNAc-Conjugated Splice-Switching ASOs to Non-Hepatic Cells through Ectopic Expression of Asialoglycoprotein Receptor

Scharner J, Qi S, Rigo F, Bennett CF, Krainer AR

Molecular Therapy Nucleic Acids

Targeting an RNA-Binding Protein Network in Acute Myeloid Leukemia

Wang E, Lu SX, Pastore A, Chen X, Imig J, Chun-Wei Lee S, Hockemeyer K, Ghebrechristos YE, Yoshimi A, Inoue D, Ki M, Cho H, Bitner L, Kloetgen A, Lin KT, Uehara T, Owa T, Tibes R, Krainer AR, Abdel-Wahab O, Aifantis I

Cancer Cell

2018

Quantitative Activity Profile and Context Dependence of All Human 5' Splice Sites

Wong MS, Kinney JB, Krainer AR

Molecular Cell

[CSHL Press Release]

Antisense Oligonucleotides Correct the Familial Dysautonomia Splicing Defect in IKBKAP Transgenic Mice

Sinha R, Kim YJ, Nomakuchi T, Sahashi K, Hua Y, Rigo F, Bennett CF, Krainer AR

Nucleic Acids Research

[CSHL Press Release]

Mechanism of Nonsense-Mediated mRNA Decay Stimulation by Splicing Factor SRSF1

Aznarez I, Nomakuchi T, Tetenbaum-Novatt J, Rahman MA, Fregoso O, Rees H, Krainer AR