AAV9 Vector: a Novel modality in gene therapy for spinal muscular atrophy

scientific article published on 26 June 2019

AAV9 Vector: a Novel modality in gene therapy for spinal muscular atrophy is …
instance of (P31):
scholarly articleQ13442814
review articleQ7318358

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P356DOI10.1038/S41434-019-0085-4
P698PubMed publication ID31243392

P2093author name stringXue-Jun Li
Yongchao Mou
Rithu Pattali
P2860cites workAn assembly chaperone collaborates with the SMN complex to generate spliceosomal SnRNPsQ24311843
A novel nuclear structure containing the survival of motor neurons proteinQ24324247
Progresses towards safe and efficient gene therapy vectorsQ26781432
Antisense oligonucleotides: treating neurodegeneration at the level of RNAQ27007056
Skeletal muscle DNA damage precedes spinal motor neuron DNA damage in a mouse model of Spinal Muscular Atrophy (SMA)Q27312220
Spinal Muscular AtrophyQ28080774
LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1Q28210584
Pan-ethnic carrier screening and prenatal diagnosis for spinal muscular atrophy: clinical laboratory analysis of >72,400 specimensQ28244756
Systemic restoration of UBA1 ameliorates disease in spinal muscular atrophyQ28279022
Early heart failure in the SMNDelta7 model of spinal muscular atrophy and correction by postnatal scAAV9-SMN deliveryQ28288188
The SMN complex: an assembly machine for RNPsQ28294580
Identification and characterization of a spinal muscular atrophy-determining geneQ29547495
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 atrophyQ30651854
Adeno-associated virus serotypes: vector toolkit for human gene therapyQ33249130
Ribonucleoprotein assembly defects correlate with spinal muscular atrophy severity and preferentially affect a subset of spliceosomal snRNPsQ33300324
Intravenous administration of self-complementary AAV9 enables transgene delivery to adult motor neuronsQ33713769
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 modelQ34030144
Assays for the identification and prioritization of drug candidates for spinal muscular atrophyQ34082155
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Antisense oligonucleotides delivered to the mouse CNS ameliorate symptoms of severe spinal muscular atrophyQ34168139
Systemic gene delivery in large species for targeting spinal cord, brain, and peripheral tissues for pediatric disordersQ34205620
Molecular therapeutic strategies for spinal muscular atrophies: current and future clinical trials.Q34393566
The survival motor neuron protein in spinal muscular atrophyQ34435656
Spinal muscular atrophy: a time for screeningQ34623310
Self-complementary AAV vectors; advances and applicationsQ34804660
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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 primatesQ35153080
Dysfunction of axonal membrane conductances in adolescents and young adults with spinal muscular atrophyQ35540059
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 systemQ35902332
SMN1 and SMN2 copy numbers in cell lines derived from patients with spinal muscular atrophy as measured by array digital PCRQ35907736
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 atrophyQ36292754
Results from a phase 1 study of nusinersen (ISIS-SMN(Rx)) in children with spinal muscular atrophyQ36661721
SMN deficiency causes tissue-specific perturbations in the repertoire of snRNAs and widespread defects in splicingQ36746476
Chaperoning ribonucleoprotein biogenesis in health and diseaseQ36777301
Plastin-3 extends survival and reduces severity in mouse models of spinal muscular atrophyQ37677406
Cardiac pathology in spinal muscular atrophy: a systematic reviewQ37748085
Spinal muscular atrophy: going beyond the motor neuronQ38066335
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 developmentQ38188560
Neurodegeneration in spinal muscular atrophy: from disease phenotype and animal models to therapeutic strategies and beyondQ38197609
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 atrophyQ39293468
Lentivector-mediated SMN replacement in a mouse model of spinal muscular atrophy.Q40480552
Replication competent helper functions for recombinant AAV vector generationQ40705869
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 atrophyQ41102726
RETRACTED ARTICLE: Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMNQ41275236
Early onset muscle weakness and disruption of muscle proteins in mouse models of spinal muscular atrophyQ41913343
Survival motor neuron protein reduction deregulates autophagy in spinal cord motoneurons in vitroQ42056056
Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytesQ42739394
Systemic delivery of scAAV9 expressing SMN prolongs survival in a model of spinal muscular atrophyQ44329621
Recombinant adenovirus expressing adeno-associated virus cap and rep proteins supports production of high-titer recombinant adeno-associated virusQ45736928
A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiencyQ45857796
A brief account of viral vectors and their promise for gene therapyQ45863967
Intravenous scAAV9 delivery of a codon-optimized SMN1 sequence rescues SMA miceQ45865368
Single-Dose Gene-Replacement Therapy for Spinal Muscular AtrophyQ45873849
Europe gives gene therapy the green lightQ45883214
Emerging Issues in AAV-Mediated In Vivo Gene TherapyQ47554736
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 patientQ52605306
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 testingQ84362191
P433issue7-8
P921main subjectspinal muscular atrophyQ580290
muscular atrophyQ2844600
P1104number of pages9
P304page(s)287-295
P577publication date2019-06-26
P1433published inGene TherapyQ15763095
P1476titleAAV9 Vector: a Novel modality in gene therapy for spinal muscular atrophy
P478volume26