| review article | Q7318358 |
| scholarly article | Q13442814 |
| P356 | DOI | 10.1038/MT.2008.171 |
| P698 | PubMed publication ID | 18682697 |
| P2093 | author name string | Douglas M McCarty | |
| P2860 | cites work | Packaging capacity of adeno-associated virus serotypes: impact of larger genomes on infectivity and postentry steps | Q24533013 |
| Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy | Q24535690 | ||
| Safety and efficacy of gene transfer for Leber's congenital amaurosis | Q24634724 | ||
| Transduction with recombinant adeno-associated virus for gene therapy is limited by leading-strand synthesis | Q24684374 | ||
| Self-complementary recombinant adeno-associated virus (scAAV) vectors promote efficient transduction independently of DNA synthesis | Q28213353 | ||
| Effect of gene therapy on visual function in Leber's congenital amaurosis | Q28277981 | ||
| Infectious clones and vectors derived from adeno-associated virus (AAV) serotypes other than AAV type 2. | Q33781976 | ||
| Nonrandom transduction of recombinant adeno-associated virus vectors in mouse hepatocytes in vivo: cell cycling does not influence hepatocyte transduction | Q33802664 | ||
| Recruitment of single-stranded recombinant adeno-associated virus vector genomes and intermolecular recombination are responsible for stable transduction of liver in vivo | Q33811409 | ||
| DNA helicase-mediated packaging of adeno-associated virus type 2 genomes into preformed capsids | Q33951407 | ||
| The AAV origin binding protein Rep68 is an ATP-dependent site-specific endonuclease with DNA helicase activity | Q34186060 | ||
| RGD inclusion in VP3 provides adeno-associated virus type 2 (AAV2)-based vectors with a heparan sulfate-independent cell entry mechanism | Q34194223 | ||
| Rapid and highly efficient transduction by double-stranded adeno-associated virus vectors in vitro and in vivo | Q34277742 | ||
| Virus-mediated transduction of murine retina with adeno-associated virus: effects of viral capsid and genome size. | Q34342913 | ||
| Endosomal processing limits gene transfer to polarized airway epithelia by adeno-associated virus. | Q34433170 | ||
| Host cell DNA repair pathways in adeno-associated viral genome processing | Q35139314 | ||
| Second-strand genome conversion of adeno-associated virus type 2 (AAV-2) and AAV-5 is not rate limiting following apical infection of polarized human airway epithelia | Q35150199 | ||
| Efficient transduction of skeletal muscle using vectors based on adeno-associated virus serotype 6. | Q45571369 | ||
| Adeno-associated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivo | Q45709105 | ||
| The packaging capacity of adeno-associated virus (AAV) and the potential for wild-type-plus AAV gene therapy vectors | Q45763222 | ||
| Quantitative analysis of the packaging capacity of recombinant adeno-associated virus | Q45766162 | ||
| Significantly increased lifespan and improved behavioral performances by rAAV gene delivery in adult mucopolysaccharidosis IIIB mice | Q45867578 | ||
| Ocular gene transfer with self-complementary AAV vectors | Q45869032 | ||
| AAV vector-mediated reversal of hypoglycemia in canine and murine glycogen storage disease type Ia. | Q45876170 | ||
| Expanding AAV packaging capacity with trans-splicing or overlapping vectors: a quantitative comparison | Q45880308 | ||
| Self-complementary recombinant adeno-associated viral vectors: packaging capacity and the role of rep proteins in vector purity. | Q50695335 | ||
| Retrograde viral delivery of IGF-1 prolongs survival in a mouse ALS model. | Q51659718 | ||
| Efficient mouse airway transduction following recombination between AAV vectors carrying parts of a larger gene. | Q51712353 | ||
| Single-stranded DNA-protein interactions in canine parvovirus. | Q52538924 | ||
| Looking into the safety of AAV vectors | Q59076157 | ||
| Efficient retrograde neuronal transduction utilizing self-complementary AAV1 | Q64376808 | ||
| Intraneural colchicine inhibition of adenoviral and adeno-associated viral vector remote spinal cord gene delivery | Q64378097 | ||
| AAV loves an active genome | Q73588694 | ||
| AAV vectors, insertional mutagenesis, and cancer | Q81326066 | ||
| Safe and efficient transduction of the liver after peripheral vein infusion of self-complementary AAV vector results in stable therapeutic expression of human FIX in nonhuman primates | Q35628868 | ||
| Self-complementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman primate liver | Q35848563 | ||
| Purification of host cell enzymes involved in adeno-associated virus DNA replication | Q35857727 | ||
| Adeno-associated virus type 2-mediated transduction of murine hematopoietic cells with long-term repopulating ability and sustained expression of a human globin gene in vivo | Q35881204 | ||
| Adeno-associated virus type 2-mediated transduction in primary human bone marrow-derived CD34+ hematopoietic progenitor cells: donor variation and correlation of transgene expression with cellular differentiation. | Q35898060 | ||
| Existence of transient functional double-stranded DNA intermediates during recombinant AAV transduction | Q35928797 | ||
| Integration of adeno-associated virus (AAV) and recombinant AAV vectors | Q35965997 | ||
| Adeno-associated virus (AAV) vectors in the CNS. | Q36173003 | ||
| Hematopoietic stem cell transduction by recombinant adeno-associated virus vectors: problems and solutions | Q36184772 | ||
| Complete in vitro reconstitution of adeno-associated virus DNA replication requires the minichromosome maintenance complex proteins | Q36424141 | ||
| Rapid uncoating of vector genomes is the key to efficient liver transduction with pseudotyped adeno-associated virus vectors | Q36509889 | ||
| Serotype-dependent packaging of large genes in adeno-associated viral vectors results in effective gene delivery in mice. | Q36545533 | ||
| Versatility of AAV vectors for retinal gene transfer | Q36965152 | ||
| Adeno-associated viral vectors and the retina | Q37055045 | ||
| AAV-mediated gene therapy for retinal disorders: from mouse to man. | Q37140652 | ||
| Targeting gene expression to cones with human cone opsin promoters in recombinant AAV. | Q37304864 | ||
| Adenovirus-facilitated nuclear translocation of adeno-associated virus type 2. | Q39685806 | ||
| Single-polarity recombinant adeno-associated virus 2 vector-mediated transgene expression in vitro and in vivo: mechanism of transduction | Q40034184 | ||
| Optimization of self-complementary AAV vectors for liver-directed expression results in sustained correction of hemophilia B at low vector dose | Q40039645 | ||
| Adeno-associated virus mediated gene transfer into lung cancer cells promoting CD40 ligand-based immunotherapy | Q40098391 | ||
| Differential internalization and nuclear uncoating of self-complementary adeno-associated virus pseudotype vectors as determinants of cardiac cell transduction | Q40111373 | ||
| Major subsets of human dendritic cells are efficiently transduced by self-complementary adeno-associated virus vectors 1 and 2. | Q40168483 | ||
| Adeno-associated virus 2-mediated gene transfer: role of a cellular serine/threonine protein phosphatase in augmenting transduction efficiency | Q40205113 | ||
| Mechanisms of AAV transduction in glaucoma-associated human trabecular meshwork cells | Q40310606 | ||
| Genomic stability of self-complementary adeno-associated virus 2 during early stages of transduction in mouse muscle in vivo | Q40376079 | ||
| Enhanced transduction of mouse bone marrow-derived dendritic cells by repetitive infection with self-complementary adeno-associated virus 6 combined with immunostimulatory ligands | Q40379085 | ||
| Adeno-associated virus serotypes 1 to 5 mediated tumor cell directed gene transfer and improvement of transduction efficiency | Q40411792 | ||
| Self-complementary adeno-associated virus 2 (AAV)-T cell protein tyrosine phosphatase vectors as helper viruses to improve transduction efficiency of conventional single-stranded AAV vectors in vitro and in vivo | Q40499221 | ||
| Adeno-associated virus vectors integrate at chromosome breakage sites | Q40543184 | ||
| Role of viral vectors and virion shells in cellular gene expression | Q40581135 | ||
| AAV serotype 2 vectors preferentially integrate into active genes in mice | Q40643545 | ||
| Transduction of hepatocellular carcinoma (HCC) using recombinant adeno-associated virus (rAAV): in vitro and in vivo effects of genotoxic agents | Q40868185 | ||
| Rapid, widespread transduction of the murine myocardium using self-complementary Adeno-associated virus | Q41909668 | ||
| High-level transgene expression in nonhuman primate liver with novel adeno-associated virus serotypes containing self-complementary genomes | Q42424212 | ||
| Effects of gamma irradiation on the transduction of dividing and nondividing cells in brain and muscle of rats by adeno-associated virus vectors | Q42525047 | ||
| Effects of adeno-associated virus DNA hairpin structure on recombination | Q42755152 | ||
| Delivery of MDR1 small interfering RNA by self-complementary recombinant adeno-associated virus vector | Q43198030 | ||
| Enhancement of gene transfer with recombinant adeno-associated virus (rAAV) vectors into primary B-cell chronic lymphocytic leukemia cells by CpG-oligodeoxynucleotides | Q43593730 | ||
| Self-complementary adeno-associated virus serotype 2 vector: global distribution and broad dispersion of AAV-mediated transgene expression in mouse brain | Q44686125 | ||
| Recombinant self-complementary adeno-associated virus serotype vector-mediated hematopoietic stem cell transduction and lineage-restricted, long-term transgene expression in a murine serial bone marrow transplantation model. | Q45396180 | ||
| Stable integration of recombinant adeno-associated virus vector genomes after transduction of murine hematopoietic stem cells. | Q45396874 | ||
| Adeno-associated virus of a single-polarity DNA genome is capable of transduction in vivo | Q45397958 | ||
| Neonatal intraperitoneal or intravenous injections of recombinant adeno-associated virus type 8 transduce dorsal root ganglia and lower motor neurons | Q45398907 | ||
| Assessment of ocular transduction using single-stranded and self-complementary recombinant adeno-associated virus serotype 2/8. | Q45399272 | ||
| Early, sustained efficacy of adeno-associated virus vector-mediated gene therapy in glycogen storage disease type Ia. | Q45417049 | ||
| Evaluation of primitive murine hematopoietic stem and progenitor cell transduction in vitro and in vivo by recombinant adeno-associated virus vector serotypes 1 through 5. | Q45418745 | ||
| Adeno-associated virus serotype 8 efficiently delivers genes to muscle and heart | Q45507049 | ||
| P433 | issue | 10 | |
| P304 | page(s) | 1648-1656 | |
| P577 | publication date | 2008-08-05 | |
| P1433 | published in | Molecular Therapy | Q15762400 |
| P1476 | title | Self-complementary AAV vectors; advances and applications | |
| P478 | volume | 16 |
| Q42429977 | A network comprising short and long noncoding RNAs and RNA helicase controls mouse retina architecture |
| Q30408313 | A potassium leak channel silences hyperactive neurons and ameliorates status epilepticus |
| Q36996899 | A reliable and feasible qPCR strategy for titrating AAV vectors |
| Q92186150 | AAV GCG-EGFP, a new tool to identify glucagon-secreting α-cells |
| Q90135526 | AAV Gene Therapy Prevents and Reverses Heart Failure in a Murine Knockout Model of Barth Syndrome |
| Q40173850 | AAV Gene Therapy for Alcoholism: Inhibition of Mitochondrial Aldehyde Dehydrogenase Enzyme Expression in Hepatoma Cells |
| Q37498000 | AAV Vectors Vaccines Against Infectious Diseases. |
| Q38585967 | AAV mediated GDNF secretion from retinal glia slows down retinal degeneration in a rat model of retinitis pigmentosa |
| Q37346460 | AAV retinal transduction in a large animal model species: comparison of a self-complementary AAV2/5 with a single-stranded AAV2/5 vector. |
| Q27318266 | AAV-Delivered Antibody Mediates Significant Protective Effects against SIVmac239 Challenge in the Absence of Neutralizing Activity |
| Q92666846 | AAV-PHP.B Administration Results in a Differential Pattern of CNS Biodistribution in Non-human Primates Compared with Mice |
| Q40052760 | AAV-PHP.B-Mediated Global-Scale Expression in the Mouse Nervous System Enables GBA1 Gene Therapy for Wide Protection from Synucleinopathy |
| Q47556217 | AAV-mediated gene delivery of the calreticulin anti-angiogenic domain inhibits ocular neovascularization |
| Q38236204 | AAV-mediated gene therapy for atherosclerosis |
| Q27009291 | AAV-mediated gene therapy in mouse models of recessive retinal degeneration |
| Q30573515 | AAV1.NT-3 gene therapy for charcot-marie-tooth neuropathy |
| Q93053748 | AAV9 Vector: a Novel modality in gene therapy for spinal muscular atrophy |
| Q57562597 | AAVvector-mediated in vivo reprogramming into pluripotency |
| Q92345084 | Adeno-Associated Viral Vectors in Neuroscience Research |
| Q39409371 | Adeno-Associated Virus (AAV) as a Vector for Gene Therapy. |
| Q26782605 | Adeno-associated Virus as a Mammalian DNA Vector |
| Q30885494 | Adeno-associated viral vectors and their redirection to cell-type specific receptors |
| Q34621723 | Adeno-associated virus vectors serotype 2 induce prolonged proliferation of capsid-specific CD8+ T cells in mice. |
| Q37272261 | Adeno-associated virus-mediated gene therapy for metabolic myopathy |
| Q34459999 | Adeno-associated virus: a key to the human genome? |
| Q41931475 | Advancements in the design and scalable production of viral gene transfer vectors. |
| Q39613077 | An adeno-associated virus vector efficiently and specifically transduces mouse skeletal muscle |
| Q35658856 | Antibody-based protection against HIV infection by vectored immunoprophylaxis |
| Q41443741 | Biophysical and ultrastructural characterization of adeno-associated virus capsid uncoating and genome release |
| Q40595052 | Blocking sense-strand activity improves potency, safety and specificity of anti-hepatitis B virus short hairpin RNA. |
| Q37690476 | CD8+ T cell recognition of epitopes within the capsid of adeno-associated virus 8-based gene transfer vectors depends on vectors' genome. |
| Q30493891 | CNS-targeted gene therapy improves survival and motor function in a mouse model of spinal muscular atrophy |
| Q93047556 | CRISPR-READI: Efficient Generation of Knockin Mice by CRISPR RNP Electroporation and AAV Donor Infection |
| Q35656153 | Capsid Mutated Adeno-Associated Virus Delivered to the Anterior Chamber Results in Efficient Transduction of Trabecular Meshwork in Mouse and Rat |
| Q38345989 | Cartilage tissue engineering: recent advances and perspectives from gene regulation/therapy |
| Q42574125 | Concepts and Strategies in Retinal Gene Therapy. |
| Q41630799 | Construction and gene expression analysis of a single-stranded DNA minivector based on an inverted terminal repeat of adeno-associated virus |
| Q36968803 | Crystallization and preliminary X-ray structural studies of adeno-associated virus serotype 6 |
| Q38827606 | Current and future prospects for hemophilia gene therapy |
| Q27002627 | Current prospects and challenges for epilepsy gene therapy |
| Q55199271 | DNA Methylation of PTGIS Enhances Hepatic Stellate Cells Activation and Liver Fibrogenesis. |
| Q38993966 | DNA Minicircle Technology Improves Purity of Adeno-associated Viral Vector Preparations |
| Q54210517 | Development of Intrathecal AAV9 Gene Therapy for Giant Axonal Neuropathy. |
| Q35260617 | Development of viral vectors for use in cardiovascular gene therapy |
| Q39694559 | Differential effects of DNA double-strand break repair pathways on single-strand and self-complementary adeno-associated virus vector genomes |
| Q35184199 | Distinct transduction profiles in the CNS via three injection routes of AAV9 and the application to generation of a neurodegenerative mouse model. |
| Q35665070 | Effectiveness of gene delivery systems for pluripotent and differentiated cells. |
| Q41673539 | Effects of Cellular Methylation on Transgene Expression and Site-Specific Integration of Adeno-Associated Virus. |
| Q40448669 | Effects of Self-Complementarity, Codon Optimization, Transgene, and Dose on Liver Transduction with AAV8. |
| Q84980524 | Effects of recombinant adeno-associated viral vectors on angiopoiesis and osteogenesis in cultured rabbit bone marrow stem cells via co-expressing hVEGF and hBMP genes: A preliminary study in vitro |
| Q34898118 | Efficiency and safety of AAV-mediated gene delivery of the human ND4 complex I subunit in the mouse visual system |
| Q40235080 | Efficient CNS targeting in adult mice by intrathecal infusion of single-stranded AAV9-GFP for gene therapy of neurological disorders |
| Q40363171 | Efficient Gene Delivery and Expression in Pancreas and Pancreatic Tumors by Capsid-Optimized AAV8 Vectors. |
| Q64913270 | Efficient Gene Transfer to the Central Nervous System by Single-Stranded Anc80L65. |
| Q45872026 | Efficient central nervous system AAVrh10-mediated intrathecal gene transfer in adult and neonate rats |
| Q41768455 | Efficient expression of self-complementary AAV in ganglion cells of the ex vivo primate retina |
| Q47554736 | Emerging Issues in AAV-Mediated In Vivo Gene Therapy |
| Q37213826 | Engraftment of a galactose receptor footprint onto adeno-associated viral capsids improves transduction efficiency |
| Q90017264 | Enhanced CRISPR-Cas9 correction of Duchenne muscular dystrophy in mice by a self-complementary AAV delivery system |
| Q41572928 | Enhanced recognition and neutralization of HIV-1 by antibody-derived CCR5-mimetic peptide variants |
| Q34407786 | Establishment of a reverse genetics system for studying human bocavirus in human airway epithelia |
| Q34656341 | Evaluation of lateral spread of transgene expression following subretinal AAV-mediated gene delivery in dogs |
| Q40080450 | Gene Delivery Approaches for Mesenchymal Stem Cell Therapy: Strategies to Increase Efficiency and Specificity |
| Q98292031 | Gene Therapy Preserves Retinal Structure and Function in a Mouse Model of NMNAT1-Associated Retinal Degeneration |
| Q89993188 | Gene Therapy for ALS-A Perspective |
| Q91734216 | Gene Therapy for Neurologic Disease: A Neurosurgical Review |
| Q40070708 | Gene Therapy in the Nervous System: Failures and Successes. |
| Q51745116 | Gene Therapy via Trans-Splicing for LMNA-Related Congenital Muscular Dystrophy. |
| Q40445294 | Gene Transfer in Isolated Adult Cardiomyocytes |
| Q38306886 | Gene delivery in tissue engineering and regenerative medicine. |
| Q30514129 | Gene delivery to mitochondria by targeting modified adenoassociated virus suppresses Leber's hereditary optic neuropathy in a mouse model. |
| Q33917592 | Gene delivery with viral vectors for cerebrovascular diseases |
| Q98564773 | Gene editing and elimination of latent herpes simplex virus in vivo |
| Q64914212 | Gene suppressing therapy for Pelizaeus-Merzbacher disease using artificial microRNA. |
| Q47558790 | Gene therapy for Mucopolysaccharidoses. |
| Q57677438 | Gene therapy for Rett syndrome: prospects and challenges |
| Q35884548 | Gene therapy for dyslipidemia: a review of gene replacement and gene inhibition strategies |
| Q45873452 | Gene therapy for inherited retinal and optic nerve degenerations. |
| Q33881920 | Gene therapy for monogenic liver diseases: clinical successes, current challenges and future prospects. |
| Q36698258 | Gene therapy for the prevention of vein graft disease |
| Q38053151 | Gene therapy in animal models of autosomal dominant retinitis pigmentosa |
| Q36147481 | Gene therapy of inherited retinopathies: a long and successful road from viral vectors to patients |
| Q38907972 | Gene therapy to rescue retinal degeneration caused by mutations in rhodopsin. |
| Q37684597 | Gene transfer of arginine kinase to skeletal muscle using adeno-associated virus |
| Q83155819 | Genetic therapy for spinal muscular atrophy |
| Q34706913 | Genetic vaccination for re-establishing T-cell tolerance in type 1 diabetes |
| Q42123243 | Hairpin-end conformation of adeno-associated virus genome determines interactions with DNA-repair pathways |
| Q38076953 | Hemophilia clinical gene therapy: brief review |
| Q35568513 | Hepatorenal correction in murine glycogen storage disease type I with a double-stranded adeno-associated virus vector |
| Q42448457 | Herpes simplex virus type 1/adeno-associated virus hybrid vectors |
| Q33713361 | High-efficiency transduction of the mouse retina by tyrosine-mutant AAV serotype vectors |
| Q33713945 | High-efficiency transient transduction of human embryonic stem cell-derived neurons with baculoviral vectors |
| Q51824910 | Highly effective ex vivo gene manipulation to study kidney development using self-complementary adenoassociated viruses. |
| Q38530599 | Highly efficient homology-driven genome editing in human T cells by combining zinc-finger nuclease mRNA and AAV6 donor delivery. |
| Q36582838 | Host Anti-antibody Responses Following Adeno-associated Virus-mediated Delivery of Antibodies Against HIV and SIV in Rhesus Monkeys |
| Q40141920 | Human Bocavirus 1 Is a Novel Helper for Adeno-Associated Virus Replication. |
| Q94502776 | Human Immune Responses to Adeno-Associated Virus (AAV) Vectors |
| Q45875226 | Human Retinal Explant Culture for Ex Vivo Validation of AAV Gene Therapy. |
| Q38183539 | Hydrodynamic transfection for generation of novel mouse models for liver cancer research |
| Q35836406 | Identification and characterization of human nucleus pulposus cell specific serotypes of adeno-associated virus for gene therapeutic approaches of intervertebral disc disorders |
| Q37366610 | Improved Adeno-associated Viral Gene Transfer to Murine Glioma. |
| Q45876893 | In search of proof-of-concept: gene therapy for glycogen storage disease type Ia. |
| Q35806350 | In vitro characterization of the activity of PF-05095808, a novel biological agent for hepatitis C virus therapy |
| Q39493298 | Inhibiting the oncogenic mir-221 by microRNA sponge: toward microRNA-based therapeutics for hepatocellular carcinoma |
| Q37939843 | Innate Immune Responses to AAV Vectors |
| Q33622746 | Inner limiting membrane barriers to AAV-mediated retinal transduction from the vitreous |
| Q40942242 | Intracerebroventricular delivery of self-complementary adeno-associated virus serotype 9 to the adult rat brain. |
| Q40375925 | Intracranial AAV-IFN-β gene therapy eliminates invasive xenograft glioblastoma and improves survival in orthotopic syngeneic murine model |
| Q33713769 | Intravenous administration of self-complementary AAV9 enables transgene delivery to adult motor neurons |
| Q45865368 | Intravenous scAAV9 delivery of a codon-optimized SMN1 sequence rescues SMA mice |
| Q35720673 | Inverse zonation of hepatocyte transduction with AAV vectors between mice and non-human primates. |
| Q34495453 | Leber's Hereditary Optic Neuropathy-Gene Therapy: From Benchtop to Bedside |
| Q28831343 | Ligand-binding domains of nuclear receptors facilitate tight control of split CRISPR activity |
| Q37784661 | Lighting a candle in the dark: advances in genetics and gene therapy of recessive retinal dystrophies |
| Q41925391 | Liver-directed gene therapy for murine glycogen storage disease type Ib. |
| Q91636324 | Long-Term Delivery of an Anti-SIV Monoclonal Antibody With AAV |
| Q35546692 | Long-term RNA interference gene therapy in a dominant retinitis pigmentosa mouse model |
| Q36169589 | Long-term, efficient inhibition of microRNA function in mice using rAAV vectors |
| Q39126013 | Manufacturing of recombinant adeno-associated viruses using mammalian expression platforms |
| Q28973610 | Massively parallel cis-regulatory analysis in the mammalian central nervous system |
| Q37007749 | Microglia-specific targeting by novel capsid-modified AAV6 vectors |
| Q27007782 | Molecular, genetic and stem cell-mediated therapeutic strategies for spinal muscular atrophy (SMA) |
| Q89762313 | Mucopolysaccharidosis Type II: One Hundred Years of Research, Diagnosis, and Treatment |
| Q37254889 | NADH-dehydrogenase type-2 suppresses irreversible visual loss and neurodegeneration in the EAE animal model of MS. |
| Q27690725 | New tools for investigating astrocyte-to-neuron communication |
| Q22306293 | Oligonucleotide therapeutic approaches for Huntington disease |
| Q37737844 | PABPN1 gene therapy for oculopharyngeal muscular dystrophy |
| Q30358434 | Passive immunization against HIV/AIDS by antibody gene transfer. |
| Q36401085 | Patterns of scAAV vector insertion associated with oncogenic events in a mouse model for genotoxicity |
| Q38012709 | Phoenix rising: gene therapy makes a comeback |
| Q38818422 | Plasmodium meets AAV-the (un)likely marriage of parasitology and virology, and how to make the match |
| Q64091291 | Precision Medicine and Exercise Therapy in Duchenne Muscular Dystrophy |
| Q35750005 | Preclinical dose-finding study with a liver-tropic, recombinant AAV-2/8 vector in the mouse model of galactosialidosis |
| Q38245677 | Preclinical toxicity evaluation of AAV for pain: evidence from human AAV studies and from the pharmacology of analgesic drugs |
| Q38988629 | Probing the Link among Genomic Cargo, Contact Mechanics, and Nanoindentation in Recombinant Adeno-Associated Virus 2. |
| Q42575413 | Production and titering of recombinant adeno-associated viral vectors |
| Q37621530 | Promise and problems associated with the use of recombinant AAV for the delivery of anti-HIV antibodies |
| Q28596492 | Proof of concept study with an HER-2 mimotope anticancer vaccine deduced from a novel AAV-mimotope library platform |
| Q39351028 | RNAi-mediated Gene Silencing of Mutant Myotilin Improves Myopathy in LGMD1A Mice. |
| Q36114209 | Rapid transgene expression in multiple precursor cell types of adult rat subventricular zone mediated by adeno-associated type 1 vectors. |
| Q37678176 | Recent advances in lentiviral vector development and applications |
| Q36058890 | Recombinant AAV Vectors for Enhanced Expression of Authentic IgG. |
| Q35078590 | Recombinant AAV-directed gene therapy for type I glycogen storage diseases |
| Q38592185 | Recombinant Human Myelin-Associated Glycoprotein Promoter Drives Selective AAV-Mediated Transgene Expression in Oligodendrocytes. |
| Q35819157 | Recombinant adeno-associated viral vector production and purification. |
| Q30837462 | Redirecting N-acetylaspartate metabolism in the central nervous system normalizes myelination and rescues Canavan disease. |
| Q40697697 | Resolving Adeno-Associated Viral Particle Diversity With Charge Detection Mass Spectrometry. |
| Q59353018 | Retinal AAV8-RS1 Gene Therapy for X-Linked Retinoschisis: Initial Findings from a Phase I/IIa Trial by Intravitreal Delivery |
| Q47551580 | Safety and Efficacy of AAV Retrograde Pancreatic Ductal Gene Delivery in Normal and Pancreatic Cancer Mice |
| Q34700126 | Safety and effects of the vector for the Leber hereditary optic neuropathy gene therapy clinical trial. |
| Q35972733 | Self-Complementary Adeno-Associated Virus Vectors Improve Transduction Efficiency of Corneal Endothelial Cells |
| Q35144378 | Self-complementary AAV2.5-BMP2-coated femoral allografts mediated superior bone healing versus live autografts in mice with equivalent biomechanics to unfractured femur |
| Q42012887 | Several rAAV vectors efficiently cross the blood-brain barrier and transduce neurons and astrocytes in the neonatal mouse central nervous system |
| Q38809600 | Short DNA Hairpins Compromise Recombinant Adeno-Associated Virus Genome Homogeneity. |
| Q40445332 | Silencing Genes in the Heart |
| Q40099591 | Single stranded adeno-associated virus achieves efficient gene transfer to anterior segment in the mouse eye. |
| Q94528057 | Small Alphaherpesvirus Latency-Associated Promoters Drive Efficient and Long-Term Transgene Expression in the CNS |
| Q91622135 | Soluble Heparin Binding Epidermal Growth Factor-Like Growth Factor Is a Regulator of GALGT2 Expression and GALGT2-Dependent Muscle and Neuromuscular Phenotypes |
| Q30828907 | Somatic Therapy of a Mouse SMA Model with a U7 snRNA Gene Correcting SMN2 Splicing |
| Q38692453 | Somatic correction of junctional epidermolysis bullosa by a highly recombinogenic AAV variant. |
| Q91044827 | Split intein-mediated selection of cells containing two plasmids using a single antibiotic |
| Q91652467 | Sustained Correction of a Murine Model of Phenylketonuria following a Single Intravenous Administration of AAVHSC15-PAH |
| Q35874710 | Sustained correction of OTC deficiency in spf( ash) mice using optimized self-complementary AAV2/8 vectors. |
| Q90538931 | Synaptic Specificity and Application of Anterograde Trans-Synaptic AAV for Probing Neural Circuitry |
| Q41471833 | Systemic delivery of tyrosine-mutant AAV vectors results in robust transduction of neurons in adult mice |
| Q37481531 | Tailored transgene expression to specific cell types in the central nervous system after peripheral injection with AAV9. |
| Q39181050 | Targeted delivery of microRNA-29b by transferrin-conjugated anionic lipopolyplex nanoparticles: a novel therapeutic strategy in acute myeloid leukemia |
| Q37878086 | Targeted gene therapy for the treatment of heart failure |
| Q92489485 | Targeting microglia with lentivirus and AAV: Recent advances and remaining challenges |
| Q64039743 | The Alpha-1-Antitrypsin Promoter Improves the Efficacy of an AAV Vector for the Treatment of MNGIE |
| Q35067592 | The genome of self-complementary adeno-associated viral vectors increases Toll-like receptor 9-dependent innate immune responses in the liver |
| Q37804497 | The next step in gene delivery: Molecular engineering of adeno-associated virus serotypes |
| Q37503995 | The upstream enhancer elements of the G6PC promoter are critical for optimal G6PC expression in murine glycogen storage disease type Ia. |
| Q24655262 | Therapeutic microRNA delivery suppresses tumorigenesis in a murine liver cancer model |
| Q34233343 | Transduction of the inner mouse retina using AAVrh8 and AAVrh10 via intravitreal injection |
| Q38030978 | Update on gene and stem cell therapy approaches for spinal muscular atrophy |
| Q39447181 | Use of Adeno-Associated Virus Vector for Cardiac Gene Delivery in Large-Animal Surgical Models of Heart Failure |
| Q41909454 | Use of self-complementary adeno-associated virus serotype 2 as a tracer for labeling axons: implications for axon regeneration |
| Q64230167 | Validation of the nearest-neighbor model for Watson-Crick self-complementary DNA duplexes in molecular crowding condition |
| Q35056714 | Vector design influences hepatic genotoxicity after adeno-associated virus gene therapy. |
| Q34561531 | Vector platforms for gene therapy of inherited retinopathies. |
| Q37295534 | Vector-mediated gene transfer engenders long-lived neutralizing activity and protection against SIV infection in monkeys |
| Q38338190 | Viral expression cassette elements to enhance transgene target specificity and expression in gene therapy |
| Q37707643 | Viral expression of insulin-like growth factor I E-peptides increases skeletal muscle mass but at the expense of strength |
| Q38803350 | miR-29b supplementation decreases expression of matrix proteins and improves alveolarization in mice exposed to maternal inflammation and neonatal hyperoxia. |
| Q41920847 | microRNA-7 upregulates death receptor 5 and primes resistant brain tumors to caspase-mediated apoptosis |
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