Publications

> >>   Selected publications of David R. Hampson

>>   Early Postnatal Expression Mitigates Immune Responses to Cas9 in the Murine Central Nervous System. Molecular Therapy, (2025)  Robert Duba-Kiss and David R. Hampson (2025)    Methods and Clinical Development, 33(3):10153  DOI:  10.1016/j.omtm.2025.1

 >>> see journal Commentary on our article in this issue:

https://doi.org/10.1016/j.omtm.2025.101640

> Neonatal but not Juvenile Treatment with AAV Gene Therapy Reduces Seizures and Prolongs Lifespan in a Mouse Model of SCN1B-Dravet Syndrome. (2025) Chunling Chen, Yukun Yuan, Heather A. O'Malley, Robert Duba-Kiss, Yan Chen, Karl Habig, Yosuke Niibori, Samantha L. Hodges, David R. Hampson, and Lori L. Isom. Journal of Clinical Investigation, https://www.jci.org/articles/view/182584

>>  CNS Dominant Human FMRP Isoform Rescues Seizures, Fear, and Sleep Abnormalities in Fmr1 KO Mice. (2023) Hayes Wong, Alexander W. M. Hooper, Hye Ri Kang, Shiron J. Lee, Jiayi Zhao, Chanchal Sadhu, Satinder Rawat, Steven J. Gray, and David R. Hampson. Journal of Clinical Investigation - Insight

https://insight.jci.org/articles/view/169650

> In Silico Prediction and In Vivo Testing of Promoters Targeting GABAergic Inhibitory Neurons. (2023) Yosuke Niibori, Robert Duba-Kiss, Joseph T. Bruder, Jared B. Smith, and David R. Hampson. Molecular Therapy, Methods and Clinical Development, 28:330-343.  https://www.cell.com/molecular-therapy-family/methods/fulltext/S2329-0501(23)00009-8?_returnURL=https%3A%2F%2Flinkinghub.elsevier.com%2Fretrieve%2Fpii%2FS2329050123000098%3Fshowall%3Dtrue 

>  Sexually Divergent Mortality and Partial Phenotypic Rescue after Gene Therapy in a Mouse Model of Dravet Syndrome.  (2020)   Yosuke Niibori, Shiron J. Lee, Berge A. Minassian, and David R. Hampson (2020).  Human Gene Therapy, 31(5-6):339-351.  doi: 10.1089/hum.2019.225

https://journals.sagepub.com/doi/10.1089/hum.2019.225

>  GABAergic Gene Regulatory Elements used in Adeno-associated Viral Vectors.  (2021)   Robert Duba-Kiss, Yosuke Niibori, and David R. Hampson

https://www.frontiersin.org/journals/neurology/articles/10.3389/fneur.2021.745159/full

>  Gene therapy using an orthologue of human fragile X mental retardation protein partially rescues behavioural abnormalities and EEG activity. (2021)     Alexander H. W. Hooper, Hayes Wong, Yosuke Niibori, Rozita Abdoli, Subha Karumuthil-Melethil, Chunping Qiao, Olivier Danos, Joseph Bruder, and David R. Hampson (2021)    Molecular Therapy Methods and Clinical Development

https://www.cell.com/molecular-therapy-family/advances/fulltext/S2329-0501(21)00115-7

>  Autism Spectrum Disorders and Neuropathology of the Cerebellum.  David R. Hampson and Gene J. Blatt. (2015)  Frontiers in Neuroscience, published: 06 November 2015 doi: 10.3389/fnins.2015.00420
https://journal.frontiersin.org/article/10.3389/fnins.2015.00420

>  Sexually Divergent Mortality and Partial Phenotypic Rescue after Gene Therapy in a Mouse Model of Dravet Syndrome.  (2020)   Yosuke Niibori, Shiron J. Lee, Berge A. Minassian, and David R. Hampson (2020).  Human Gene Therapy, 31(5-6):339-351.  doi: 10.1089/hum.2019.225

>  The Application of Adeno-Associated Viral Vector Gene Therapy to the Treatment of Fragile X Syndrome.  (2019)  David R. Hampson, Alexander Hooper, and Yosuke Niibori (2019).  Brain Science  2019, 9, 32;  doi:10.3390/brainsci9020032

https://www.mdpi.com/2076-3425/9/2/32

> Interregulation between Fragile X Mental Retardation Protein and Methyl CpG Binding Protein 2 (2020) Jason Arsenault, Alexander W. M. Hooper, Shervin Gholizadeh, , Laura K Pacey, Enea Koxhioni, Yosuke Niibori, James H. Eubanks, Lu-Yang Wang, David R. Hampson (2020) . Human Molecular Genetics, 29(23): 3744-3756. doi: 10.1093/hmg/ddaa226.PMID: 33084871
>  Identification of a molecular locus for normalizing dysregulated GABA release from interneurons in the Fragile X brain.  Yang YM, Arsenault J, Bah A, Krzeminski M, Fekete A, Chao OY, Pacey LK, Wang A, Forman-Kay J, Hampson DR, Wang LY. (2018)  Molecular Psychiatry 2020 Sep 17. doi: 10.1038/s41380-018-0240-0.  PMID: 30224722

>  FMRP Expression Levels in Mouse CNS Neurons Determine Behavioral Phenotype (2016)   

Jason Arsenault, Shervin Gholizadeh, Yosuke Niibori, Laura K. Pacey, Sebok K. Halder, Enea Koxhioni, Ayumu Konno, Hirokazu Hirai, and David R. Hampson. (2016)  Human Gene Therapy 27(12), 982-996 (2016).  DOI: 10.1089/hum.2016.090

Mammalian Susceptibility to a Neonicotinoid Insecticide after Fetal and Early Postnatal Exposure.    Andrew P. Burke, Yosuke Niibori, Hayato Terayama, Masatoshi Ito, Charlotte Pidgeon, Jason Arsenault, Pablo R. Camarero, Carolyn L. Cummins, Rafael Mateo, Kou Sakabe, and David R. Hampson (2018)  Scientific Reports (Nature Publishing Group), 8 (1),16639.   https://www.nature.com/articles/s41598-018-35129-5