| scholarly article | Q13442814 |
| P50 | author | Paul N. Valdmanis | Q54282802 |
| P2093 | author name string | Mark A Kay | |
| P2860 | cites work | Biotechnology. A prudent path forward for genomic engineering and germline gene modification | Q22065510 |
| The nuclear RNase III Drosha initiates microRNA processing | Q24295234 | ||
| Gene targeting in stem cells from individuals with osteogenesis imperfecta | Q24310539 | ||
| Membrane-associated heparan sulfate proteoglycan is a receptor for adeno-associated virus type 2 virions | Q24531466 | ||
| RNA-guided human genome engineering via Cas9 | Q24598394 | ||
| Targeting DNA double-strand breaks with TAL effector nucleases | Q24605087 | ||
| Vision 1 year after gene therapy for Leber's congenital amaurosis | Q24607981 | ||
| Therapeutic targeting of microRNAs: current status and future challenges | Q38228101 | ||
| Somatic correction of junctional epidermolysis bullosa by a highly recombinogenic AAV variant. | Q38692453 | ||
| Dicer-independent processing of short hairpin RNAs | Q39200306 | ||
| RNA interference improves myopathic phenotypes in mice over-expressing FSHD region gene 1 (FRG1). | Q39319041 | ||
| In vivo post-transcriptional gene silencing of alpha-1 antitrypsin by adeno-associated virus vectors expressing siRNA. | Q40115238 | ||
| CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells | Q40486239 | ||
| A Designer AAV Variant Permits Efficient Retrograde Access to Projection Neurons | Q40532321 | ||
| Genome editing with CRISPR/Cas9 in postnatal mice corrects PRKAG2 cardiac syndrome. | Q40602766 | ||
| CNS-restricted Transduction and CRISPR/Cas9-mediated Gene Deletion with an Engineered AAV Vector | Q40643280 | ||
| Inhibition of hepatitis B virus in mice by RNA interference | Q40648698 | ||
| Human gene targeting by viral vectors | Q41480639 | ||
| Off-target-free gene delivery by affinity-purified receptor-targeted viral vectors | Q41493938 | ||
| An α-synuclein AAV gene silencing vector ameliorates a behavioral deficit in a rat model of Parkinson's disease, but displays toxicity in dopamine neurons | Q41874717 | ||
| Sustained miRNA-mediated knockdown of mutant AAT with simultaneous augmentation of wild-type AAT has minimal effect on global liver miRNA profiles. | Q42029205 | ||
| Allele-specific locus binding and genome editing by CRISPR at the p16INK4a locus | Q42397056 | ||
| RNA interference-mediated suppression and replacement of human rhodopsin in vivo | Q42413344 | ||
| RNA interference in adult mice. | Q43038702 | ||
| Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors | Q43716886 | ||
| Therapeutic benefit derived from RNAi-mediated ablation of IMPDH1 transcripts in a murine model of autosomal dominant retinitis pigmentosa (RP10). | Q44077833 | ||
| Systemic delivery of scAAV9 expressing SMN prolongs survival in a model of spinal muscular atrophy | Q44329621 | ||
| RNAi suppresses polyglutamine-induced neurodegeneration in a model of spinocerebellar ataxia. | Q44964949 | ||
| In vivo-directed evolution of a new adeno-associated virus for therapeutic outer retinal gene delivery from the vitreous | Q45350780 | ||
| Gene targeting in vivo by adeno-associated virus vectors. | Q45413871 | ||
| Permanent inactivation of Huntington's disease mutation by personalized allele-specific CRISPR/Cas9. | Q48840079 | ||
| RNA interference. Drugging RNAi. | Q55360325 | ||
| Viral delivery of miR-196a ameliorates the SBMA phenotype via the silencing of CELF2 | Q84281961 | ||
| Case 15-2009: a man with coma after cardiac arrest | Q95802924 | ||
| Multiplex genome engineering using CRISPR/Cas systems | Q24609428 | ||
| A coding-independent function of gene and pseudogene mRNAs regulates tumour biology | Q24632651 | ||
| Therapeutic microRNA delivery suppresses tumorigenesis in a murine liver cancer model | Q24655262 | ||
| A Programmable Dual-RNA-Guided DNA Endonuclease in Adaptive Bacterial Immunity | Q24669850 | ||
| A combinatorial approach to create artificial homing endonucleases cleaving chosen sequences | Q24670002 | ||
| The evolution of the DLK1-DIO3 imprinted domain in mammals | Q27333130 | ||
| Argonaute2 is the catalytic engine of mammalian RNAi | Q27860545 | ||
| Origins and Mechanisms of miRNAs and siRNAs | Q27860822 | ||
| Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans | Q27860867 | ||
| A system for stable expression of short interfering RNAs in mammalian cells | Q27860875 | ||
| Passenger-strand cleavage facilitates assembly of siRNA into Ago2-containing RNAi enzyme complexes | Q28115751 | ||
| Both natural and designed micro RNAs can inhibit the expression of cognate mRNAs when expressed in human cells | Q28207586 | ||
| Short hairpin RNAs (shRNAs) induce sequence-specific silencing in mammalian cells | Q28215061 | ||
| In vivo genome editing restores haemostasis in a mouse model of haemophilia | Q28241725 | ||
| Highly efficient endogenous human gene correction using designed zinc-finger nucleases | Q28243157 | ||
| Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial | Q28262431 | ||
| Human RISC couples microRNA biogenesis and posttranscriptional gene silencing | Q28280424 | ||
| Targeted genome engineering in human cells with the Cas9 RNA-guided endonuclease | Q28284509 | ||
| Small CRISPR RNAs guide antiviral defense in prokaryotes | Q28290898 | ||
| Reshaping human antibodies for therapy | Q28290936 | ||
| A universal system to select gene-modified hepatocytes in vivo | Q28468539 | ||
| Cas9-crRNA ribonucleoprotein complex mediates specific DNA cleavage for adaptive immunity in bacteria | Q29614420 | ||
| RNA-guided genetic silencing systems in bacteria and archaea | Q29614421 | ||
| Intervening sequences of regularly spaced prokaryotic repeats derive from foreign genetic elements | Q29615788 | ||
| Double nicking by RNA-guided CRISPR Cas9 for enhanced genome editing specificity | Q29615792 | ||
| CRISPR RNA-guided activation of endogenous human genes | Q29617072 | ||
| Clustered regularly interspaced short palindrome repeats (CRISPRs) have spacers of extrachromosomal origin | Q29617073 | ||
| Natural RNA circles function as efficient microRNA sponges | Q29617892 | ||
| Circular RNAs are a large class of animal RNAs with regulatory potency | Q29620042 | ||
| CRISPR-Cas9 knockin mice for genome editing and cancer modeling | Q30607892 | ||
| 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 | ||
| Engineering and selection of shuffled AAV genomes: a new strategy for producing targeted biological nanoparticles | Q33337378 | ||
| Therapeutic genome editing by combined viral and non-viral delivery of CRISPR system components in vivo | Q33652566 | ||
| Artificial microRNAs as siRNA shuttles: improved safety as compared to shRNAs in vitro and in vivo | Q33712990 | ||
| Nonallele-specific silencing of mutant and wild-type huntingtin demonstrates therapeutic efficacy in Huntington's disease mice. | Q33713689 | ||
| Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy. | Q36598414 | ||
| A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice | Q36673956 | ||
| Sustained secretion of human alpha-1-antitrypsin from murine muscle transduced with adeno-associated virus vectors | Q36734619 | ||
| Targeted gene addition in human CD34(+) hematopoietic cells for correction of X-linked chronic granulomatous disease | Q36777548 | ||
| Homology-driven genome editing in hematopoietic stem and progenitor cells using ZFN mRNA and AAV6 donors. | Q36828138 | ||
| RNA interference-induced hepatotoxicity results from loss of the first synthesized isoform of microRNA-122 in mice. | Q36880385 | ||
| In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy. | Q36943717 | ||
| Widespread Central Nervous System Gene Transfer and Silencing After Systemic Delivery of Novel AAV-AS Vector | Q36951265 | ||
| In vivo gene editing in dystrophic mouse muscle and muscle stem cells | Q37045850 | ||
| An essential receptor for adeno-associated virus infection. | Q37130666 | ||
| Intrastriatal rAAV-mediated delivery of anti-huntingtin shRNAs induces partial reversal of disease progression in R6/1 Huntington's disease transgenic mice | Q37131550 | ||
| Robust RNAi enhancement via human Argonaute-2 overexpression from plasmids, viral vectors and cell lines | Q37327641 | ||
| Cre-dependent selection yields AAV variants for widespread gene transfer to the adult brain | Q37382030 | ||
| Therapeutic AAV9-mediated suppression of mutant SOD1 slows disease progression and extends survival in models of inherited ALS | Q37393810 | ||
| Widespread spinal cord transduction by intrathecal injection of rAAV delivers efficacious RNAi therapy for amyotrophic lateral sclerosis | Q37463967 | ||
| Selection and evaluation of clinically relevant AAV variants in a xenograft liver model | Q37610916 | ||
| A multifunctional AAV-CRISPR-Cas9 and its host response | Q37731772 | ||
| State-of-the-art gene-based therapies: the road ahead | Q37862074 | ||
| Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges | Q37866086 | ||
| In vivo gene delivery by nonviral vectors: overcoming hurdles? | Q38004708 | ||
| RNA interference improves motor and neuropathological abnormalities in a Huntington's disease mouse model | Q33936969 | ||
| CRISPR elements in Yersinia pestis acquire new repeats by preferential uptake of bacteriophage DNA, and provide additional tools for evolutionary studies. | Q33985645 | ||
| Programmable RNA recognition and cleavage by CRISPR/Cas9. | Q34042210 | ||
| In vivo genome editing using Staphylococcus aureus Cas9 | Q34043628 | ||
| Rationally engineered Cas9 nucleases with improved specificity | Q34045530 | ||
| Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage | Q34046684 | ||
| Systematic comparison of CRISPR/Cas9 and RNAi screens for essential genes | Q34046778 | ||
| Safe, long-term hepatic expression of anti-HCV shRNA in a nonhuman primate model | Q34284247 | ||
| The loop position of shRNAs and pre-miRNAs is critical for the accuracy of dicer processing in vivo | Q34310927 | ||
| Efficacy and long-term safety of alipogene tiparvovec (AAV1-LPLS447X) gene therapy for lipoprotein lipase deficiency: an open-label trial | Q34311144 | ||
| Virus-delivered small RNA silencing sustains strength in amyotrophic lateral sclerosis | Q34313224 | ||
| A pseudogene long-noncoding-RNA network regulates PTEN transcription and translation in human cells | Q34329463 | ||
| Gene silencing using micro-RNA designed hairpins | Q34364532 | ||
| Genome-Scale CRISPR-Mediated Control of Gene Repression and Activation | Q34443093 | ||
| Chemically modified guide RNAs enhance CRISPR-Cas genome editing in human primary cells | Q34482784 | ||
| High-fidelity CRISPR-Cas9 nucleases with no detectable genome-wide off-target effects. | Q34507554 | ||
| Parallel shRNA and CRISPR-Cas9 screens enable antiviral drug target identification | Q34520015 | ||
| Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways | Q34530552 | ||
| Targeted chromosomal cleavage and mutagenesis in Drosophila using zinc-finger nucleases | Q34615432 | ||
| Artificial miRNAs mitigate shRNA-mediated toxicity in the brain: implications for the therapeutic development of RNAi | Q34768688 | ||
| Long-term safety and efficacy of factor IX gene therapy in hemophilia B. | Q34783922 | ||
| Terminal N-linked galactose is the primary receptor for adeno-associated virus 9. | Q34787342 | ||
| Promoterless gene targeting without nucleases ameliorates haemophilia B in mice. | Q34979802 | ||
| Delayed disease onset and extended survival in the SOD1G93A rat model of amyotrophic lateral sclerosis after suppression of mutant SOD1 in the motor cortex | Q34983540 | ||
| Adeno-associated virus gene repair corrects a mouse model of hereditary tyrosinemia in vivo. | Q35105022 | ||
| Efficient production of Fah-null heterozygote pigs by chimeric adeno-associated virus-mediated gene knockout and somatic cell nuclear transfer. | Q35249273 | ||
| AAV6-mediated systemic shRNA delivery reverses disease in a mouse model of facioscapulohumeral muscular dystrophy | Q35568525 | ||
| In vivo interrogation of gene function in the mammalian brain using CRISPR-Cas9 | Q35820051 | ||
| Alleviation of off-target effects from vector-encoded shRNAs via codelivered RNA decoys | Q35910008 | ||
| Hepatic gene transfer in neonatal mice by adeno-associated virus serotype 8 vector. | Q35988663 | ||
| In Vivo Selection Yields AAV-B1 Capsid for Central Nervous System and Muscle Gene Therapy | Q35999976 | ||
| RNA interference inhibits DUX4-induced muscle toxicity in vivo: implications for a targeted FSHD therapy. | Q36085352 | ||
| New recombinant serotypes of AAV vectors. | Q36172991 | ||
| Broadening the targeting range of Staphylococcus aureus CRISPR-Cas9 by modifying PAM recognition | Q36398694 | ||
| P433 | issue | 4 | |
| P921 | main subject | gene therapy | Q213901 |
| P304 | page(s) | 361-372 | |
| P577 | publication date | 2017-01-10 | |
| P1433 | published in | Human Gene Therapy | Q15757580 |
| P1476 | title | Future of rAAV Gene Therapy: Platform for RNAi, Gene Editing, and Beyond | |
| P478 | volume | 28 |
| Q39196756 | AAV: An Overview of Unanswered Questions |
| Q57492577 | Adeno-Associated Virus-Mediated RNAi against Mutant Alleles Attenuates Abnormal Calvarial Phenotypes in an Apert Syndrome Mouse Model |
| Q89602953 | Cancer gene therapy by NF-κB-activated cancer cell-specific expression of CRISPR/Cas9 targeting telomeres |
| Q91272447 | Cancer therapy with a CRISPR-assisted telomerase-activating gene expression system |
| Q93081159 | Cell and Gene Therapies for Mucopolysaccharidoses: Base Editing and Therapeutic Delivery to the CNS |
| Q98906651 | FGF/FGFR signaling in health and disease |
| Q45873745 | Immunity to CRISPR Cas9 and Cas12a therapeutics |
| Q47594709 | Methods and Applications of CRISPR-Mediated Base Editing in Eukaryotic Genomes |
| Q89932125 | MicroRNA-based recombinant AAV vector assembly improves efficiency of suicide gene transfer in a murine model of lymphoma |
| Q59792605 | Trisomy silencing by XIST normalizes Down syndrome cell pathogenesis demonstrated for hematopoietic defects in vitro |
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