| scholarly article | Q13442814 |
| P356 | DOI | 10.1517/14712598.2012.711306 |
| P698 | PubMed publication ID | 22849423 |
| P2093 | author name string | Nicholas M Boulis | |
| Eleanor M Donnelly | |||
| P2860 | cites work | Gemin3: A novel DEAD box protein that interacts with SMN, the spinal muscular atrophy gene product, and is a component of gems | Q22010941 |
| Gemin4. A novel component of the SMN complex that is found in both gems and nucleoli | Q22253358 | ||
| Gemin5, a novel WD repeat protein component of the SMN complex that binds Sm proteins | Q24291913 | ||
| Gemin8 is a novel component of the survival motor neuron complex and functions in small nuclear ribonucleoprotein assembly | Q24301969 | ||
| Intragenic telSMN mutations: frequency, distribution, evidence of a founder effect, and modification of the spinal muscular atrophy phenotype by cenSMN copy number. | Q24539804 | ||
| A SMN missense mutation complements SMN2 restoring snRNPs and rescuing SMA mice | Q24657021 | ||
| Unrip is a component of SMN complexes active in snRNP assembly | Q28246568 | ||
| The SMN complex: an assembly machine for RNPs | Q28294580 | ||
| A role for SMN exon 7 splicing in the selective vulnerability of motor neurons in spinal muscular atrophy | Q28586960 | ||
| SMN in spinal muscular atrophy and snRNP biogenesis | Q29013847 | ||
| Identification and characterization of a spinal muscular atrophy-determining gene | Q29547495 | ||
| In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector | Q29547503 | ||
| Induced pluripotent stem cells from a spinal muscular atrophy patient | Q29614340 | ||
| Immunogenicity of induced pluripotent stem cells | Q29616185 | ||
| A single nucleotide in the SMN gene regulates splicing and is responsible for spinal muscular atrophy | Q29617367 | ||
| Neural stem cell transplantation can ameliorate the phenotype of a mouse model of spinal muscular atrophy | Q30483218 | ||
| CNS-targeted gene therapy improves survival and motor function in a mouse model of spinal muscular atrophy | Q30493891 | ||
| Ribonucleoprotein assembly defects correlate with spinal muscular atrophy severity and preferentially affect a subset of spliceosomal snRNPs | Q33300324 | ||
| AAV's anatomy: roadmap for optimizing vectors for translational success | Q33659389 | ||
| Mouse survival motor neuron alleles that mimic SMN2 splicing and are inducible rescue embryonic lethality early in development but not late | Q33797677 | ||
| Codon bias and heterologous protein expression | Q33979754 | ||
| Clinical protocol. Gene therapy of Canavan disease: AAV-2 vector for neurosurgical delivery of aspartoacylase gene (ASPA) to the human brain | Q34142832 | ||
| Temporal requirement for high SMN expression in SMA mice | Q34192528 | ||
| The survival motor neuron protein in spinal muscular atrophy | Q34435656 | ||
| Selective and rapid uptake of adeno-associated virus type 2 in brain | Q34471578 | ||
| Treatment of late infantile neuronal ceroid lipofuscinosis by CNS administration of a serotype 2 adeno-associated virus expressing CLN2 cDNA. | Q34778098 | ||
| Self-complementary AAV vectors; advances and applications | Q34804660 | ||
| Knockdown of the survival motor neuron (Smn) protein in zebrafish causes defects in motor axon outgrowth and pathfinding. | Q35567371 | ||
| Bifunctional RNAs targeting the intronic splicing silencer N1 increase SMN levels and reduce disease severity in an animal model of spinal muscular atrophy | Q35665141 | ||
| Direct central nervous system delivery provides enhanced protection following vector mediated gene replacement in a severe model of spinal muscular atrophy | Q35675298 | ||
| Adeno-associated virus (AAV) vectors in the CNS. | Q36173003 | ||
| A transgene carrying an A2G missense mutation in the SMN gene modulates phenotypic severity in mice with severe (type I) spinal muscular atrophy. | Q36323111 | ||
| Inactivation of the survival motor neuron gene, a candidate gene for human spinal muscular atrophy, leads to massive cell death in early mouse embryos | Q36578997 | ||
| SMN deficiency causes tissue-specific perturbations in the repertoire of snRNAs and widespread defects in splicing | Q36746476 | ||
| Chaperoning ribonucleoprotein biogenesis in health and disease | Q36777301 | ||
| Deciphering the assembly pathway of Sm-class U snRNPs | Q37113000 | ||
| Delivery of bifunctional RNAs that target an intronic repressor and increase SMN levels in an animal model of spinal muscular atrophy | Q37154271 | ||
| Efficient transfer, integration, and sustained long-term expression of the transgene in adult rat brains injected with a lentiviral vector | Q37251114 | ||
| SMN-assisted assembly of snRNP-specific Sm cores in trypanosomes | Q37272307 | ||
| Survival motor neuron protein modulates neuron-specific apoptosis | Q37292520 | ||
| Cervical spinal cord therapeutics delivery: preclinical safety validation of a stabilized microinjection platform | Q37393215 | ||
| Stem cell technology for the study and treatment of motor neuron diseases. | Q37851503 | ||
| Translating stem cell studies to the clinic for CNS repair: current state of the art and the need for a Rosetta stone | Q37879267 | ||
| A mouse model for spinal muscular atrophy | Q38316939 | ||
| Cervical multilevel intraspinal stem cell therapy: assessment of surgical risks in Gottingen minipigs | Q39628535 | ||
| Lentivector-mediated SMN replacement in a mouse model of spinal muscular atrophy. | Q40480552 | ||
| Disruption of an SF2/ASF-dependent exonic splicing enhancer in SMN2 causes spinal muscular atrophy in the absence of SMN1. | Q40741164 | ||
| Correlation between severity and SMN protein level in spinal muscular atrophy | Q41102726 | ||
| RETRACTED ARTICLE: Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN | Q41275236 | ||
| Deletion of smn-1, the Caenorhabditis elegans ortholog of the spinal muscular atrophy gene, results in locomotor dysfunction and reduced lifespan | Q41977041 | ||
| Reduced U snRNP assembly causes motor axon degeneration in an animal model for spinal muscular atrophy | Q42548444 | ||
| The human centromeric survival motor neuron gene (SMN2) rescues embryonic lethality in Smn(-/-) mice and results in a mouse with spinal muscular atrophy | Q43569750 | ||
| Rabies virus glycoprotein pseudotyping of lentiviral vectors enables retrograde axonal transport and access to the nervous system after peripheral delivery | Q43685007 | ||
| Prevalence of serum IgG and neutralizing factors against adeno-associated virus (AAV) types 1, 2, 5, 6, 8, and 9 in the healthy population: implications for gene therapy using AAV vectors | Q44258968 | ||
| Systemic delivery of scAAV9 expressing SMN prolongs survival in a model of spinal muscular atrophy | Q44329621 | ||
| Neuromuscular defects in a Drosophila survival motor neuron gene mutant. | Q44463867 | ||
| Intravenous scAAV9 delivery of a codon-optimized SMN1 sequence rescues SMA mice | Q45865368 | ||
| Characterization of a murine model of SMA. | Q45876097 | ||
| The Caenorhabditis elegans orthologue of the human gene responsible for spinal muscular atrophy is a maternal product critical for germline maturation and embryonic viability. | Q47068856 | ||
| Progressive and selective degeneration of motoneurons in a mouse model of SMA. | Q47786186 | ||
| Targeted spinal cord therapeutics delivery: stabilized platform and microelectrode recording guidance validation | Q48030830 | ||
| Stimulating full-length SMN2 expression by delivering bifunctional RNAs via a viral vector. | Q54610896 | ||
| P433 | issue | 11 | |
| P407 | language of work or name | English | Q1860 |
| P921 | main subject | spinal muscular atrophy | Q580290 |
| P1104 | number of pages | 9 | |
| P304 | page(s) | 1463-1471 | |
| P577 | publication date | 2012-07-31 | |
| P1433 | published in | Expert Opinion on Biological Therapy | Q5421201 |
| P1476 | title | Update on gene and stem cell therapy approaches for spinal muscular atrophy | |
| P478 | volume | 12 |
| Q89477851 | Drug treatment for spinal muscular atrophy types II and III | cites work | P2860 |
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