| P50 | author | Damien J Cabral | Q55362943 |
| P2093 | author name string | Qin Su | |
| | Douglas R Martin | |
| | Heather L Gray-Edwards | |
| | Ellen Sapp | |
| | Marian DiFiglia | |
| | Neil Aronin | |
| | Guangping Gao | |
| | Miguel Sena-Esteves | |
| | Allison M Keeler | |
| | Aime K Johnson | |
| | Jacob A Johnson | |
| | Lorelei Stoica | |
| | Sourav R Choudhury | |
| | Anne F Harris | |
| | Jennifer S Ferreira | |
| P2860 | cites work | Huntingtin is a cytoplasmic protein associated with vesicles in human and rat brain neurons | Q24317574 |
| | The atomic structure of adeno-associated virus (AAV-2), a vector for human gene therapy. | Q24533492 |
| | Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy | Q24535690 |
| | Adeno-associated virus type 2 VP2 capsid protein is nonessential and can tolerate large peptide insertions at its N terminus | Q24563214 |
| | Directed evolution of adeno-associated virus to an infectious respiratory virus | Q24649385 |
| | Analysis of Relative Gene Expression Data Using Real-Time Quantitative PCR and the 2−ΔΔCT Method | Q25938999 |
| | Structural Insight into the Unique Properties of Adeno-Associated Virus Serotype 9 | Q27678476 |
| | A novel gene containing a trinucleotide repeat that is expanded and unstable on Huntington's disease chromosomes. | Q27860836 |
| | A single intravenous rAAV injection as late as P20 achieves efficacious and sustained CNS Gene therapy in Canavan mice. | Q30559496 |
| | Neuronal targets for reducing mutant huntingtin expression to ameliorate disease in a mouse model of Huntington's disease | Q30581321 |
| | Sustained normalization of neurological disease after intracranial gene therapy in a feline model | Q30644848 |
| | Random peptide libraries displayed on adeno-associated virus to select for targeted gene therapy vectors | Q30975099 |
| | Glial promoter selectivity following AAV-delivery to the immature brain | Q31120301 |
| | Image-guided convection-enhanced delivery platform in the treatment of neurological diseases. | Q31141544 |
| | Several rAAV vectors efficiently cross the blood-brain barrier and transduce neurons and astrocytes in the neonatal mouse central nervous system | Q42012887 |
| | Correction of neurological disease of mucopolysaccharidosis IIIB in adult mice by rAAV9 trans-blood-brain barrier gene delivery | Q42059455 |
| | Targeted gene delivery to the enteric nervous system using AAV: a comparison across serotypes and capsid mutants. | Q42174892 |
| | Intracerebral administration of adeno-associated viral vector serotype rh.10 carrying human SGSH and SUMF1 cDNAs in children with mucopolysaccharidosis type IIIA disease: results of a phase I/II trial | Q42228547 |
| | Intracerebroventricular injection of adeno-associated virus 6 and 9 vectors for cell type-specific transgene expression in the spinal cord | Q42257686 |
| | Long-term gene expression and phenotypic correction using adeno-associated virus vectors in the mammalian brain | Q42280576 |
| | Eight years of clinical improvement in MPTP-lesioned primates after gene therapy with AAV2-hAADC. | Q42502571 |
| | Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes | Q42739394 |
| | A single injection of an adeno-associated virus vector into nuclei with divergent connections results in widespread vector distribution in the brain and global correction of a neurogenetic disease | Q44453459 |
| | Effective gene therapy for an inherited CNS disease in a large animal model | Q44875065 |
| | Recombinant AAV viral vectors pseudotyped with viral capsids from serotypes 1, 2, and 5 display differential efficiency and cell tropism after delivery to different regions of the central nervous system | Q45007196 |
| | Gene therapy for aromatic L-amino acid decarboxylase deficiency | Q45215332 |
| | Enhanced survival of the LINCL mouse following CLN2 gene transfer using the rh.10 rhesus macaque-derived adeno-associated virus vector. | Q45408947 |
| | Recombinant adeno-associated virus purification using novel methods improves infectious titer and yield | Q45747699 |
| | Directed evolution of adeno-associated virus yields enhanced gene delivery vectors | Q33232270 |
| | In vitro and in vivo gene therapy vector evolution via multispecies interbreeding and retargeting of adeno-associated viruses | Q33327474 |
| | A myocardium tropic adeno-associated virus (AAV) evolved by DNA shuffling and in vivo selection | Q33411254 |
| | Molecular signatures of disease brain endothelia provide new sites for CNS-directed enzyme therapy | Q33502598 |
| | Efficient gene delivery and selective transduction of glial cells in the mammalian brain by AAV serotypes isolated from nonhuman primates. | Q33713108 |
| | Nonallele-specific silencing of mutant and wild-type huntingtin demonstrates therapeutic efficacy in Huntington's disease mice. | Q33713689 |
| | Intravenous administration of self-complementary AAV9 enables transgene delivery to adult motor neurons | Q33713769 |
| | Engineering liver-detargeted AAV9 vectors for cardiac and musculoskeletal gene transfer | Q33833473 |
| | Global CNS transduction of adult mice by intravenously delivered rAAVrh.8 and rAAVrh.10 and nonhuman primates by rAAVrh.10 | Q33870720 |
| | RNA interference improves motor and neuropathological abnormalities in a Huntington's disease mouse model | Q33936969 |
| | Systemic gene delivery in large species for targeting spinal cord, brain, and peripheral tissues for pediatric disorders | Q34205620 |
| | A phase I study of aromatic L-amino acid decarboxylase gene therapy for Parkinson's disease | Q34209755 |
| | Sustained therapeutic reversal of Huntington's disease by transient repression of huntingtin synthesis. | Q34283652 |
| | Long-term follow-up after gene therapy for canavan disease. | Q34318553 |
| | Intravenous AAV9 efficiently transduces myenteric neurons in neonate and juvenile mice | Q34341997 |
| | Results from a phase I safety trial of hAADC gene therapy for Parkinson disease | Q34656075 |
| | Artificial miRNAs mitigate shRNA-mediated toxicity in the brain: implications for the therapeutic development of RNAi | Q34768688 |
| | Treatment of late infantile neuronal ceroid lipofuscinosis by CNS administration of a serotype 2 adeno-associated virus expressing CLN2 cDNA. | Q34778098 |
| | Terminal N-linked galactose is the primary receptor for adeno-associated virus 9. | Q34787342 |
| | The AAV9 receptor and its modification to improve in vivo lung gene transfer in mice | Q35015807 |
| | Preclinical differences of intravascular AAV9 delivery to neurons and glia: a comparative study of adult mice and nonhuman primates | Q35086714 |
| | Functional analysis of the putative integrin recognition motif on adeno-associated virus 9. | Q35121258 |
| | Identification of the galactose binding domain of the adeno-associated virus serotype 9 capsid | Q36155479 |
| | Production and characterization of adeno-associated viral vectors | Q36779416 |
| | Efficient gene therapy-based method for the delivery of therapeutics to primate cortex | Q37114888 |
| | Adeno-associated virus type 6 is retrogradely transported in the non-human primate brain. | Q37374468 |
| | Therapeutic AAV9-mediated suppression of mutant SOD1 slows disease progression and extends survival in models of inherited ALS | Q37393810 |
| | Drawing a high-resolution functional map of adeno-associated virus capsid by massively parallel sequencing | Q37614775 |
| | Intrajugular vein delivery of AAV9-RNAi prevents neuropathological changes and weight loss in Huntington's disease mice. | Q37696030 |
| | Correction of pathological accumulation of glycosaminoglycans in central nervous system and peripheral tissues of MPSIIIA mice through systemic AAV9 gene transfer | Q39566137 |
| | Adeno-associated virus vectors serotyped with AAV8 capsid are more efficient than AAV-1 or -2 serotypes for widespread gene delivery to the neonatal mouse brain | Q40329324 |
| | Convection-enhanced delivery of AAV vector in parkinsonian monkeys; in vivo detection of gene expression and restoration of dopaminergic function using pro-drug approach | Q40870377 |
| | RETRACTED ARTICLE: Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN | Q41275236 |
| | Off-target-free gene delivery by affinity-purified receptor-targeted viral vectors | Q41493938 |
| | Axonal transport of adeno-associated viral vectors is serotype-dependent | Q41919978 |
| P4510 | describes a project that uses | ImageJ | Q1659584 |
| P433 | issue | 4 | |
| P921 | main subject | gene silencing | Q1431332 |
| P304 | page(s) | 726-735 | |
| P577 | publication date | 2015-12-28 | |
| P1433 | published in | Molecular Therapy | Q15762400 |
| P1476 | title | Widespread Central Nervous System Gene Transfer and Silencing After Systemic Delivery of Novel AAV-AS Vector | |
| P478 | volume | 24 | |