RNAStructuromeDB: a transcriptome-wide database of predicted RNA secondary structures with integrated APIs for functional annotation and RNA-targeted drug discovery. [PDF]
Dapour AO +9 more
europepmc +1 more source
Implementation of SMA carrier testing in genetic laboratories: comparison of two methods for quantifying theSMN1gene [PDF]
Ivon Cuscó +3 more
openalex +1 more source
DNA damage/p53, innate immune, and unfolded protein responses are activated in primate liver after toxic, high-dose AAV-SMN1 delivery. [PDF]
Moeini P +8 more
europepmc +1 more source
Clinical diagnosis and genetic analysis of a rare case of Duchenne muscular dystrophy and spinal muscular atrophy. [PDF]
Liu Y +5 more
europepmc +1 more source
Efficacy and biodistribution analysis of intracerebroventricular administration of an optimized scAAV9-SMN1 vector in a mouse model of spinal muscular atrophy [PDF]
Nicole Armbruster +11 more
openalex +1 more source
Plastin 3 is upregulated in iPSC-derived motoneurons from asymptomatic SMN1-deleted individuals
Ludwig Heesen +17 more
openalex +2 more sources
Survival of motor neuron 1 telomeric (SMN1); survival of motor neuron 2 centromeric (SMN2); stasimon (STAS; CG8408) [PDF]
openalex +1 more source
Orthogonal characterization of rAAV reveals vector attributes that drive ITR repair, self-complementary genome formation, and transgene expression. [PDF]
Llinares C +10 more
europepmc +1 more source
Relationship Between Smn1 and Casp3 Gene Expression in Kainate-Induced Spinal Cord Injury Model
Nor Alia Abd Aziz +6 more
openalex +1 more source
Anterior Horn Cell Disease in Adulthood: Unmasking Spinal Muscular Atrophy Type 4. [PDF]
Rafique S, Odeh A.
europepmc +1 more source

