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1
Gene therapy for hemophilia: Progress to date and challenges moving forward.
Gollomp, Kandace L;Doshi, Bhavya S;Arruda, Valder R;
2019 Transfusion and apheresis science : official journ... DOI: S1473-0502(19)30157-... 262 views
2
Adeno-Associated Virus Technologies and Methods for Targeted Neuronal Manipulation.
Haery, Leila;Deverman, Benjamin E;Matho, Katherine S;Cetin, Ali;Woodard, Kenton;Cepko, Connie;Guerin...
2019 frontiers in neuroanatomy DOI: 10.3389/fnana.2019.0... 225 views
3
Peripheral ProBDNF Delivered by an AAV Vector to the Muscle Triggers Depression-Like Behaviours in Mice.
Lin, L Y;Kelliny, S;Liu, L C;Al-Hawwas, M;Zhou, X F;Bobrovskaya, L;
2020 neurotoxicity research DOI: 10.1007/s12640-020-0... 207 views
4
Lab-Scale Production of Recombinant Adeno-Associated Viruses (AAV) for Expression of Optogenetic Elements.
Haar, Janina;Krämer, Chiara;Grimm, Dirk;
2020 methods in molecular biology (clifton, nj) DOI: 10.1007/978-1-0716-0... 232 views
5
an amplified promoter system for targeted expression of calcium indicator proteins in the cerebellar cortex
;Bernd eKuhn;Bernd eKuhn;Bernd eKuhn;Ilker eOzden;Ilker eOzden;Ilker eOzden;Yulia eLampi;Yulia eLamp...
2012 Fish physiology and biochemistry DOI: 10.3389/fncir.2012.0... 167 views
6
non-clinical safety and efficacy of an aav2/8 vector administered intravenously for treatment of mucopolysaccharidosis type vi
;Rita Ferla;Marialuisa Alliegro;Jean-Brice Marteau;Margherita Dell’Anno;Edoardo Nusco;Severine Pouil...
2017 international journal of zoology DOI: 10.1016/j.omtm.2017.... 226 views
7
editorial: gene therapy for the central and peripheral nervous system
;Andrew P. Tosolini;George M. Smith
2018 international journal of nanomedicine DOI: 10.3389/fnmol.2018.0... 185 views
8
current challenges and future directions in recombinant aav-mediated gene therapy of duchenne muscular dystrophy
;Shin'ichi Takeda;Takashi Okada
2013 journal of cerebral blood flow and metabolism DOI: 10.3390/ph6070813 235 views
9
intracisternal delivery of nfkappab-inducible scaav2/9 reveals locoregional neuroinflammation induced by systemic kainic acid treatment.
;Olivier eBockstael;Liliane eTenenbaum;Deniz eDalkara;Catherine eMelas;Olivier eDe Witte;Marc eLeviv...
2014 international journal of nanomedicine DOI: 10.3389/fnmol.2014.0... 191 views
10
crispr/cas9-aav mediated knock-in at nrl locus in human embryonic stem cells
;Xianglian Ge;Haitao Xi;Fayu Yang;Xiao Zhi;Yanghua Fu;Ding Chen;Ren-He Xu;Ge Lin;Jia Qu;Junzhao Zhao...
2016 coordination chemistry reviews DOI: 10.1038/mtna.2016.10... 207 views
11
evaluating efficiencies of dual aav approaches for retinal targeting
;Livia S. Carvalho;Livia S. Carvalho;Livia S. Carvalho;Heikki T. Turunen;Heikki T. Turunen;Heikki T....
2017 Journal of enzyme inhibition and medicinal chemist... DOI: 10.3389/fnins.2017.0... 178 views
12
emerging issues in aav-mediated in vivo gene therapy
;Pasqualina Colella;Giuseppe Ronzitti;Federico Mingozzi
2018 international journal of zoology DOI: 10.1016/j.omtm.2017.... 178 views
13
simultaneous visualization of extrinsic and intrinsic axon collaterals in golgi-like detail for mouse corticothalamic and corticocortical cells: a double viral infection method
;Akiya eWatakabe;Akiya eWatakabe;Masafumi eTakaji;Shigeki eKato;Kazuto eKobayashi;Hiroaki eMizukami;...
2014 Fish physiology and biochemistry DOI: 10.3389/fncir.2014.0... 164 views

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gene therapy hemophilia neuroscience gene delivery cell-type specificity intersectional methods targeted neuronal manipulation viral vectors virus technologies depression anti-probdnf antibody behavioural tests golgi stain probdnf gene expression adeno-associated virus cns ultracentrifugation optogenetics Transduction