| P50 | author | Mojgan Bandehpour | Q59818634 |
| | Afrooz Rashnonejad | Q62096232 |
| P2093 | author name string | B Durmaz | |
| | H Onay | |
| | C Gündüz | |
| | F Özkınay | |
| | S Y Süslüer | |
| P2860 | cites work | Genome engineering using the CRISPR-Cas9 system | Q22122027 |
| | Viral vectors: a look back and ahead on gene transfer technology | Q26823087 |
| | Genetic correction of human induced pluripotent stem cells from patients with spinal muscular atrophy | Q27328843 |
| | Studies on transformation of Escherichia coli with plasmids | Q27860598 |
| | Co-regulation of survival of motor neuron (SMN) protein and its interactor SIP1 during development and in spinal muscular atrophy | Q28200174 |
| | CNS-targeted gene therapy improves survival and motor function in a mouse model of spinal muscular atrophy | Q30493891 |
| | Ten years of gene targeting: targeted mouse mutants, from vector design to phenotype analysis | Q33650414 |
| | Gene targeting for gene therapy: prospects | Q33755010 |
| | A single nucleotide difference that alters splicing patterns distinguishes the SMA gene SMN1 from the copy gene SMN2. | Q33864982 |
| | Spinal muscular atrophy: mechanisms and therapeutic strategies | Q33871311 |
| | Gene correction in human embryonic and induced pluripotent stem cells: promises and challenges ahead | Q33930103 |
| | Gene correction by homologous recombination with zinc finger nucleases in primary cells from a mouse model of a generic recessive genetic disease | Q33930132 |
| | Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model | Q34030144 |
| | Gene targeting of Gemin2 in mice reveals a correlation between defects in the biogenesis of U snRNPs and motoneuron cell death | Q34099265 |
| | Intramuscular scAAV9-SMN injection mediates widespread gene delivery to the spinal cord and decreases disease severity in SMA mice. | Q34321271 |
| | Design, engineering, and characterization of zinc finger nucleases | Q34559924 |
| | Genetic conversion of an SMN2 gene to SMN1: a novel approach to the treatment of spinal muscular atrophy | Q34725351 |
| | The mechanism of gene targeting in human somatic cells | Q35139201 |
| | Nuclear entry of nonviral vectors | Q35490588 |
| | Decreasing disease severity in symptomatic, Smn(-/-);SMN2(+/+), spinal muscular atrophy mice following scAAV9-SMN delivery | Q35823950 |
| | Homologous recombination and RecA protein: towards a new generation of tools for genome manipulations | Q36015367 |
| | Targeted modification of mammalian genomes | Q36144755 |
| | Intracellular trafficking of plasmids for gene therapy: mechanisms of cytoplasmic movement and nuclear import | Q36683376 |
| | Spinal muscular atrophy and a model for survival of motor neuron protein function in axonal ribonucleoprotein complexes | Q37033848 |
| | Progress and prospects: nuclear import of nonviral vectors | Q37702093 |
| | Gene therapy: a promising approach to treating spinal muscular atrophy. | Q38213245 |
| | Long-term and efficient expression of human β-globin gene in a hematopoietic cell line using a new site-specific integrating non-viral system | Q38892183 |
| | A novel single step double positive double negative selection strategy for beta-globin gene replacement. | Q40283887 |
| | Lentivector-mediated SMN replacement in a mouse model of spinal muscular atrophy. | Q40480552 |
| | Natural history in proximal spinal muscular atrophy. Clinical analysis of 445 patients and suggestions for a modification of existing classifications | Q40986555 |
| | RETRACTED ARTICLE: Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN | Q41275236 |
| | Translational fidelity of intrathecal delivery of self-complementary AAV9-survival motor neuron 1 for spinal muscular atrophy | Q42232357 |
| | Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes | Q42739394 |
| | Development of a gene therapy strategy for the restoration of survival motor neuron protein expression: implications for spinal muscular atrophy therapy | Q45859031 |
| | Intravenous scAAV9 delivery of a codon-optimized SMN1 sequence rescues SMA mice | Q45865368 |
| | Survival motor neuron SMN1 and SMN2 gene promoters: identical sequences and differential expression in neurons and non-neuronal cells | Q47431781 |
| | Construction of a plasmid containing human SMN, the SMA determining gene, coupled to EGFP. | Q53972480 |
| | Stimulating full-length SMN2 expression by delivering bifunctional RNAs via a viral vector. | Q54610896 |
| | In Vitro Restoration of Functional SMN Protein in Human Trophoblast Cells Affected by Spinal Muscular Atrophy by Small Fragment Homologous Replacement | Q58923973 |
| | International SMA consortium meeting. (26-28 June 1992, Bonn, Germany) | Q67481178 |
| | Classification of spinal muscular atrophies | Q72423037 |
| P433 | issue | 1 | |
| P921 | main subject | spinal muscular atrophy | Q580290 |
| | muscular atrophy | Q2844600 |
| P304 | page(s) | 10-17 | |
| P577 | publication date | 2015-09-02 | |
| P1433 | published in | Gene Therapy | Q15763095 |
| P1476 | title | In vitro gene manipulation of spinal muscular atrophy fibroblast cell line using gene-targeting fragment for restoration of SMN protein expression | |
| P478 | volume | 23 | |