Chowdhury EA, Meno-Tetang G, Chang HY, Wu S, Huang HW, Jamier T, et al. Current progress and limitations of AAV mediated delivery of protein therapeutic genes and the importance of developing quantitative pharmacokinetic/pharmacodynamic (PK/PD) models. Adv Drug Deliv Rev. 2021;170:214–37.
CAS PubMed Article Google Scholar
Merkel SF, Andrews AM, Lutton EM, Mu D, Hudry E, Hyman BT, et al. Trafficking of adeno-associated virus vectors across a model of the blood-brain barrier; a comparative study of transcytosis and transduction using primary human brain endothelial cells. J Neurochem. 2017;140:216–30.
CAS PubMed Article Google Scholar
Bailey RM, Rozenberg A, Gray SJ. Comparison of high-dose intracisterna magna and lumbar puncture intrathecal delivery of AAV9 in mice to treat neuropathies. Brain Res. 2020;1739:146832.
CAS PubMed PubMed Central Article Google Scholar
Murrey DA, Naughton BJ, Duncan FJ, Meadows AS, Ware TA, Campbell KJ, et al. Feasibility and safety of systemic rAAV9-hNAGLU delivery for treating mucopolysaccharidosis IIIB: toxicology, biodistribution, and immunological assessments in primates. Hum Gene Ther Clin Dev. 2014;25:72–84.
CAS PubMed PubMed Central Article Google Scholar
Mendell JR, Al-Zaidy S, Shell R, Arnold WD, Rodino-Klapac LR, Prior TW, et al. Single-dose gene-replacement therapy for spinal muscular atrophy. N Engl J Med. 2017;377:1713–22.
CAS PubMed Article Google Scholar
Mathiesen SN, Lock JL, Schoderboeck L, Abraham WC, Hughes SM. CNS transduction benefits of AAV-PHP.eB over AAV9 are dependent on administration route and mouse strain. Mol Ther Methods Clin Dev. 2020;19:447–58.
CAS PubMed PubMed Central Article Google Scholar
Chan KY, Jang MJ, Yoo BB, Greenbaum A, Ravi N, Wu WL, et al. Engineered AAVs for efficient noninvasive gene delivery to the central and peripheral nervous systems. Nat Neurosci. 2017;20:1172–9.
CAS PubMed PubMed Central Article Google Scholar
Mattar CN, Wong AM, Hoefer K, Alonso-Ferrero ME, Buckley SM, Howe SJ, et al. Systemic gene delivery following intravenous administration of AAV9 to fetal and neonatal mice and late-gestation nonhuman primates. FASEB J. 2015;29:3876–88.
CAS PubMed PubMed Central Article Google Scholar
Gray SJ, Matagne V, Bachaboina L, Yadav S, Ojeda SR, Samulski RJ. Preclinical differences of intravascular AAV9 delivery to neurons and glia: a comparative study of adult mice and nonhuman primates. Mol Ther. 2011;19:1058–69.
CAS PubMed PubMed Central Article Google Scholar
Jackson KL, Dayton RD, Klein RL. AAV9 supports wide-scale transduction of the CNS and TDP-43 disease modeling in adult rats. Mol Ther Methods Clin Dev. 2015;2:15036.
PubMed PubMed Central Article CAS Google Scholar
Hinderer C, Katz N, Buza EL, Dyer C, Goode T, Bell P, et al. Severe toxicity in nonhuman primates and piglets following high-dose intravenous administration of an adeno-associated virus vector expressing human SMN. Hum Gene Ther. 2018;29:285–98.
CAS PubMed PubMed Central Article Google Scholar
Pulicherla N, Shen S, Yadav S, Debbink K, Govindasamy L, Agbandje-McKenna M, et al. Engineering liver-detargeted AAV9 vectors for cardiac and musculoskeletal gene transfer. Mol Ther. 2011;19:1070–8.
CAS PubMed PubMed Central Article Google Scholar
Deverman BE, Pravdo PL, Simpson BP, Kumar SR, Chan KY, Banerjee A, et al. Cre-dependent selection yields AAV variants for widespread gene transfer to the adult brain. Nat Biotechnol. 2016;34:204–9.
CAS PubMed PubMed Central Article Google Scholar
Hordeaux J, Wang Q, Katz N, Buza EL, Bell P, Wilson JM. The neurotropic properties of AAV-PHP.B are limited to C57BL/6J mice. Mol Ther. 2018;26:664–8.
CAS PubMed PubMed Central Article Google Scholar
Taghian T, Marosfoi MG, Puri AS, Cataltepe OI, King RM, Diffie EB, et al. A safe and reliable technique for CNS delivery of AAV vectors in the cisterna magna. Mol Ther. 2020;28:411–21.
CAS PubMed Article Google Scholar
Iannitti T, Scarrott JM, Likhite S, Coldicott IRP, Lewis KE, Heath PR, et al. Translating SOD1 gene silencing toward the clinic: a highly efficacious, off-target-free, and biomarker-supported strategy for fALS. Mol Ther Nucleic Acids. 2018;12:75–88.
CAS PubMed PubMed Central Article Google Scholar
Sinnett SE, Hector RD, Gadalla KKE, Heindel C, Chen D, Zaric V, et al. Improved MECP2 gene therapy extends the survival of MeCP2-null mice without apparent toxicity after intracisternal delivery. Mol Ther Methods Clin Dev. 2017;5:106–15.
CAS PubMed PubMed Central Article Google Scholar
Schuster DJ, Dykstra JA, Riedl MS, Kitto KF, Belur LR, McIvor RS, et al. Biodistribution of adeno-associated virus serotype 9 (AAV9) vector after intrathecal and intravenous delivery in mouse. Front Neuroanat. 2014;8:42.
PubMed PubMed Central Google Scholar
Hordeaux J, Dubreil L, Deniaud J, Iacobelli F, Moreau S, Ledevin M, et al. Efficient central nervous system AAVrh10-mediated intrathecal gene transfer in adult and neonate rats. Gene Ther. 2015;22:316–24.
CAS PubMed Article Google Scholar
McPhee SW, Janson CG, Li C, Samulski RJ, Camp AS, Francis J, et al. Immune responses to AAV in a phase I study for Canavan disease. J Gene Med. 2006;8:577–88.
CAS PubMed Article Google Scholar
Pietersz KL, Martier RM, Baatje MS, Liefhebber JM, Brouwers CC, Pouw SM, et al. Transduction patterns in the CNS following various routes of AAV-5-mediated gene delivery. Gene Ther. 2021;28:435–46.
CAS PubMed Article Google Scholar
Taymans JM, Vandenberghe LH, Haute CV, Thiry I, Deroose CM, Mortelmans L, et al. Comparative analysis of adeno-associated viral vector serotypes 1, 2, 5, 7, and 8 in mouse brain. Hum Gene Ther. 2007;18:195–206.
CAS PubMed Article Google Scholar
Bailey RM, Armao D, Nagabhushan Kalburgi S, Gray SJ. Development of Intrathecal AAV9 Gene Therapy for Giant Axonal Neuropathy. Mol Ther Methods Clin Dev. 2018;9:160–71.
CAS PubMed PubMed Central Article Google Scholar
Federici T, Taub JS, Baum GR, Gray SJ, Grieger JC, Matthews KA, et al. Robust spinal motor neuron transduction following intrathecal delivery of AAV9 in pigs. Gene Ther. 2012;19:852–9.
CAS PubMed Article Google Scholar
Gray SJ, Nagabhushan Kalburgi S, McCown TJ, Jude Samulski R. Global CNS gene delivery and evasion of anti-AAV-neutralizing antibodies by intrathecal AAV administration in non-human primates. Gene Ther. 2013;20:450–9.
CAS PubMed PubMed Central Article Google Scholar
Hinderer C, Bell P, Vite CH, Louboutin JP, Grant R, Bote E, et al. Widespread gene transfer in the central nervous system of cynomolgus macaques following delivery of AAV9 into the cisterna magna. Mol Ther Methods Clin Dev. 2014;1:14051.
PubMed PubMed Central Article CAS Google Scholar
Pietersz KL, Martier RM, Baatje MS, Liefhebber JM, Brouwers CC, Pouw SM, et al. Transduction patterns in the CNS following various routes of AAV-5-mediated gene delivery. Gene Ther. 2021;28:35–446.
Aschauer DF, Kreuz S, Rumpel S. Analysis of transduction efficiency, tropism and axonal transport of AAV serotypes 1, 2, 5, 6, 8 and 9 in the mouse brain. PLoS One. 2013;8:e76310.
CAS PubMed PubMed Central Article Google Scholar
Chang HY, Morrow K, Bonacquisti E, Zhang W, Shah DK. Antibody pharmacokinetics in rat brain determined using microdialysis. MAbs. 2018;10:843–53.
CAS PubMed PubMed Central Article Google Scholar
Chang HY, Wu S, Meno-Tetang G, Shah DK. A translational platform PBPK model for antibody disposition in the brain. J Pharmacokinet Pharmacodyn. 2019;46:319–38.
CAS PubMed PubMed Central Article Google Scholar
Hordeaux J, Buza EL, Dyer C, Goode T, Mitchell TW, Richman L, et al. Adeno-associated virus-induced dorsal root ganglion pathology. Hum Gene Ther. 2020;31:808–18.
CAS PubMed Article Google Scholar
High-dose AAV gene therapy deaths. Nat Biotechnol. 2020;38:910.
Matagne V, Borloz E, Ehinger Y, Saidi L, Villard L, Roux JC. Severe offtarget effects following intravenous delivery of AAV9-MECP2 in a female mouse model of Rett syndrome. Neurobiol Dis. 2021;149:105235.
CAS PubMed Article Google Scholar
Cearley CN, Wolfe JH. Transduction characteristics of adeno-associated virus vectors expressing cap serotypes 7, 8, 9, and Rh10 in the mouse brain. Mol Ther. 2006;13:528–37.
CAS PubMed Article Google Scholar
Chatterjee D, Marmion DJ, McBride JL, Manfredsson FP, Butler D, Messer A, et al. Enhanced CNS transduction from AAV.PHP.eB infusion into the cisterna magna of older adult rats compared to AAV9. Gene Ther. 2021.
Palazzi X, Pardo ID, Sirivelu MP, Newman L, Kumpf SW, Qian J, et al. Biodistribution and tolerability of AAV-PHP.B-CBh-SMN1 in wistar han rats and cynomolgus macaques reveal different toxicologic profiles. Hum Gene Ther. 2022;33:175–87.
留言 (0)