| scholarly article | Q13442814 |
| P356 | DOI | 10.1038/NBT0310-235 |
| P698 | PubMed publication ID | 20212484 |
| P2093 | author name string | Alex MacKenzie | |
| P2860 | cites work | Large-scale adeno-associated viral vector production using a herpesvirus-based system enables manufacturing for clinical studies | Q24630417 |
| Sustained improvement of spinal muscular atrophy mice treated with trichostatin A plus nutrition | Q28288824 | ||
| Determination of SMN1/SMN2 gene dosage by a quantitative genotyping platform combining capillary electrophoresis and MALDI-TOF mass spectrometry | Q33232587 | ||
| A simplified baculovirus-AAV expression vector system coupled with one-step affinity purification yields high-titer rAAV stocks from insect cells | Q33713203 | ||
| Spinal muscular atrophy: why do low levels of survival motor neuron protein make motor neurons sick? | Q33785933 | ||
| Self-complementary AAV vectors; advances and applications | Q34804660 | ||
| Neuronal SMN expression corrects spinal muscular atrophy in severe SMA mice while muscle-specific SMN expression has no phenotypic effect | Q39521045 | ||
| Proteasome inhibitors decrease AAV2 capsid derived peptide epitope presentation on MHC class I following transduction. | Q39638928 | ||
| RETRACTED ARTICLE: Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN | Q41275236 | ||
| Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes | Q42739394 | ||
| Assessment of liquid microbead arrays for the screening of newborns for spinal muscular atrophy. | Q50905572 | ||
| P433 | issue | 3 | |
| P921 | main subject | spinal muscular atrophy | Q580290 |
| muscular atrophy | Q2844600 | ||
| P304 | page(s) | 235-237 | |
| P577 | publication date | 2010-03-01 | |
| P1433 | published in | Nature Biotechnology | Q1893837 |
| P1476 | title | Genetic therapy for spinal muscular atrophy | |
| P478 | volume | 28 |
| Q97570201 | Current and emerging treatment options for spinal muscular atrophy |
| Q35683524 | Disruption of the Survival Motor Neuron (SMN) gene in pigs using ssDNA. |
| Q28243993 | Prolactin increases SMN expression and survival in a mouse model of severe spinal muscular atrophy via the STAT5 pathway. |
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