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Long-Term Therapeutic Efficacy of Intravenous AAV-Mediated Hamartin Replacement in Mouse Model of Tuberous Sclerosis Type 1.
Prabhakar S, Cheah PS, Zhang X, Zinter M, Gianatasio M, Hudry E, Bronson RT, Kwiatkowski DJ, Stemmer-Rachamimov A, Maguire CA, Sena-Esteves M, Tannous BA, Breakefield XO. Prabhakar S, et al. Mol Ther Methods Clin Dev. 2019 Aug 16;15:18-26. doi: 10.1016/j.omtm.2019.08.003. eCollection 2019 Dec 13. Mol Ther Methods Clin Dev. 2019. PMID: 31534984 Free PMC article.
Selection of an Efficient AAV Vector for Robust CNS Transgene Expression.
Hanlon KS, Meltzer JC, Buzhdygan T, Cheng MJ, Sena-Esteves M, Bennett RE, Sullivan TP, Razmpour R, Gong Y, Ng C, Nammour J, Maiz D, Dujardin S, Ramirez SH, Hudry E, Maguire CA. Hanlon KS, et al. Mol Ther Methods Clin Dev. 2019 Oct 23;15:320-332. doi: 10.1016/j.omtm.2019.10.007. eCollection 2019 Dec 13. Mol Ther Methods Clin Dev. 2019. PMID: 31788496 Free PMC article.
AAV-mediated gene delivery in a feline model of Sandhoff disease corrects lysosomal storage in the central nervous system.
Rockwell HE, McCurdy VJ, Eaton SC, Wilson DU, Johnson AK, Randle AN, Bradbury AM, Gray-Edwards HL, Baker HJ, Hudson JA, Cox NR, Sena-Esteves M, Seyfried TN, Martin DR. Rockwell HE, et al. ASN Neuro. 2015 Apr 13;7(2):1759091415569908. doi: 10.1177/1759091415569908. Print 2015 Mar-Apr. ASN Neuro. 2015. PMID: 25873306 Free PMC article.
Healing genes in the nervous system.
Breakefield XO, Sena-Esteves M. Breakefield XO, et al. Neuron. 2010 Oct 21;68(2):178-81. doi: 10.1016/j.neuron.2010.10.005. Neuron. 2010. PMID: 20955923 Free PMC article.
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