scholarly article | Q13442814 |
review article | Q7318358 |
P356 | DOI | 10.1038/S41434-019-0085-4 |
P698 | PubMed publication ID | 31243392 |
P2093 | author name string | Xue-Jun Li | |
Yongchao Mou | |||
Rithu Pattali | |||
P2860 | cites work | An assembly chaperone collaborates with the SMN complex to generate spliceosomal SnRNPs | Q24311843 |
A novel nuclear structure containing the survival of motor neurons protein | Q24324247 | ||
Progresses towards safe and efficient gene therapy vectors | Q26781432 | ||
Antisense oligonucleotides: treating neurodegeneration at the level of RNA | Q27007056 | ||
Skeletal muscle DNA damage precedes spinal motor neuron DNA damage in a mouse model of Spinal Muscular Atrophy (SMA) | Q27312220 | ||
Spinal Muscular Atrophy | Q28080774 | ||
LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1 | Q28210584 | ||
Pan-ethnic carrier screening and prenatal diagnosis for spinal muscular atrophy: clinical laboratory analysis of >72,400 specimens | Q28244756 | ||
Systemic restoration of UBA1 ameliorates disease in spinal muscular atrophy | Q28279022 | ||
Early heart failure in the SMNDelta7 model of spinal muscular atrophy and correction by postnatal scAAV9-SMN delivery | Q28288188 | ||
The SMN complex: an assembly machine for RNPs | Q28294580 | ||
Identification and characterization of a spinal muscular atrophy-determining gene | Q29547495 | ||
Requirement of enhanced Survival Motoneuron protein imposed during neuromuscular junction maturation. | Q30569780 | ||
PTEN depletion decreases disease severity and modestly prolongs survival in a mouse model of spinal muscular atrophy | Q30651854 | ||
Adeno-associated virus serotypes: vector toolkit for human gene therapy | Q33249130 | ||
Ribonucleoprotein assembly defects correlate with spinal muscular atrophy severity and preferentially affect a subset of spliceosomal snRNPs | Q33300324 | ||
Intravenous administration of self-complementary AAV9 enables transgene delivery to adult motor neurons | Q33713769 | ||
Spinal muscular atrophy: why do low levels of survival motor neuron protein make motor neurons sick? | Q33785933 | ||
Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model | Q34030144 | ||
Assays for the identification and prioritization of drug candidates for spinal muscular atrophy | Q34082155 | ||
Observational study of spinal muscular atrophy type I and implications for clinical trials. | Q34135750 | ||
Antisense oligonucleotides delivered to the mouse CNS ameliorate symptoms of severe spinal muscular atrophy | Q34168139 | ||
Systemic gene delivery in large species for targeting spinal cord, brain, and peripheral tissues for pediatric disorders | Q34205620 | ||
Molecular therapeutic strategies for spinal muscular atrophies: current and future clinical trials. | Q34393566 | ||
The survival motor neuron protein in spinal muscular atrophy | Q34435656 | ||
Spinal muscular atrophy: a time for screening | Q34623310 | ||
Self-complementary AAV vectors; advances and applications | Q34804660 | ||
Gene therapy using adeno-associated virus vectors | Q34852719 | ||
Interaction of survival of motor neuron (SMN) and HuD proteins with mRNA cpg15 rescues motor neuron axonal deficits. | Q35064376 | ||
Improving single injection CSF delivery of AAV9-mediated gene therapy for SMA: a dose-response study in mice and nonhuman primates | Q35153080 | ||
Dysfunction of axonal membrane conductances in adolescents and young adults with spinal muscular atrophy | Q35540059 | ||
Knockdown of the survival motor neuron (Smn) protein in zebrafish causes defects in motor axon outgrowth and pathfinding. | Q35567371 | ||
Systemic scAAV9 variant mediates brain transduction in newborn rhesus macaques. | Q35746222 | ||
Methods for gene transfer to the central nervous system | Q35902332 | ||
SMN1 and SMN2 copy numbers in cell lines derived from patients with spinal muscular atrophy as measured by array digital PCR | Q35907736 | ||
Gene therapy progress and prospects: development of improved lentiviral and retroviral vectors--design, biosafety, and production. | Q36186475 | ||
Survival motor neuron protein in motor neurons determines synaptic integrity in spinal muscular atrophy | Q36292754 | ||
Results from a phase 1 study of nusinersen (ISIS-SMN(Rx)) in children with spinal muscular atrophy | Q36661721 | ||
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 | ||
Plastin-3 extends survival and reduces severity in mouse models of spinal muscular atrophy | Q37677406 | ||
Cardiac pathology in spinal muscular atrophy: a systematic review | Q37748085 | ||
Spinal muscular atrophy: going beyond the motor neuron | Q38066335 | ||
At the "junction" of spinal muscular atrophy pathogenesis: the role of neuromuscular junction dysfunction in SMA disease progression. | Q38091538 | ||
Spinal muscular atrophy: a motor neuron disorder or a multi-organ disease? | Q38123249 | ||
Adeno-associated virus structural biology as a tool in vector development | Q38188560 | ||
Neurodegeneration in spinal muscular atrophy: from disease phenotype and animal models to therapeutic strategies and beyond | Q38197609 | ||
SMN control of RNP assembly: from post-transcriptional gene regulation to motor neuron disease. | Q38207417 | ||
Gene therapy: a promising approach to treating spinal muscular atrophy. | Q38213245 | ||
Spinal muscular atrophy--recent therapeutic advances for an old challenge. | Q38489298 | ||
FDA-Approved Oligonucleotide Therapies in 2017. | Q39214058 | ||
How the discovery of ISS-N1 led to the first medical therapy for spinal muscular atrophy | Q39293468 | ||
Lentivector-mediated SMN replacement in a mouse model of spinal muscular atrophy. | Q40480552 | ||
Replication competent helper functions for recombinant AAV vector generation | Q40705869 | ||
SMN gene duplication and the emergence of the SMN2 gene occurred in distinct hominids: SMN2 is unique to Homo sapiens. | Q40805995 | ||
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 | ||
Early onset muscle weakness and disruption of muscle proteins in mouse models of spinal muscular atrophy | Q41913343 | ||
Survival motor neuron protein reduction deregulates autophagy in spinal cord motoneurons in vitro | Q42056056 | ||
Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes | Q42739394 | ||
Systemic delivery of scAAV9 expressing SMN prolongs survival in a model of spinal muscular atrophy | Q44329621 | ||
Recombinant adenovirus expressing adeno-associated virus cap and rep proteins supports production of high-titer recombinant adeno-associated virus | Q45736928 | ||
A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency | Q45857796 | ||
A brief account of viral vectors and their promise for gene therapy | Q45863967 | ||
Intravenous scAAV9 delivery of a codon-optimized SMN1 sequence rescues SMA mice | Q45865368 | ||
Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy | Q45873849 | ||
Europe gives gene therapy the green light | Q45883214 | ||
Emerging Issues in AAV-Mediated In Vivo Gene Therapy | Q47554736 | ||
Oligonucleotide-mediated survival of motor neuron protein expression in CNS improves phenotype in a mouse model of spinal muscular atrophy. | Q48565771 | ||
Advances in therapy for spinal muscular atrophy: promises and challenges. | Q50052600 | ||
Impaired minor tri-snRNP assembly generates differential splicing defects of U12-type introns in lymphoblasts derived from a type I SMA patient | Q52605306 | ||
Plastin 3 Promotes Motor Neuron Axonal Growth and Extends Survival in a Mouse Model of Spinal Muscular Atrophy. | Q54253770 | ||
Technical standards and guidelines for spinal muscular atrophy testing | Q84362191 | ||
P433 | issue | 7-8 | |
P921 | main subject | spinal muscular atrophy | Q580290 |
muscular atrophy | Q2844600 | ||
P1104 | number of pages | 9 | |
P304 | page(s) | 287-295 | |
P577 | publication date | 2019-06-26 | |
P1433 | published in | Gene Therapy | Q15763095 |
P1476 | title | AAV9 Vector: a Novel modality in gene therapy for spinal muscular atrophy | |
P478 | volume | 26 |