<p>Mean SMN protein levels are higher in controls compared to patients. SMN protein levels in PBMCs did not differ significantly between patients with 2, 3, 4 or 5 <i>SMN2</i> copies (p = 0.06). Higher <i>SMN2</i> copy number is associated with higher levels of SMN protein in fibroblasts (p = 0.01). Boxplot elements represent: median (line in the middle), 1<sup>st</sup> en 3<sup>rd</sup> quartile (bottom and top of the box), highest case with 1.5 time inter-quartile range (bottom and top whisker) and outliers (dots).</p
<p>(<b>A</b>) Immunoblots of SMN and β-actin following knockdown of SR proteins in GM00232 type 1 SM...
(A) Multiple comparisons of SMN concentrations in untreated or ASO-treated groups with and without P...
<p>(A) Analysis of cell proliferation in wild-type (Control), NIH3T3-Smn<sub>RNAi</sub>, NIH3T3-SMN2...
<p>(A) SMN levels with respect to age in all subjects. (B) SMN protein levels were measured in SMA p...
BACKGROUND: Clinical trials to test safety and efficacy of drugs for patients with spinal muscular a...
<p>While SMN protein levels are lower in SMA relative to Control subjects, protein levels by SMA typ...
<p>SMN levels were analyzed by CD+ cell subtype at the 7d timepoint from Study 4. <b>A</b>: Analysis...
<p>A,C: SMN2-FL, SMN-FL and Total SMN transcripts generally increase with SMA Type. SMN-FL (A) is a ...
<p>(A) SMN2 mRNA expression levels in blood from patients with SMA. SMN2-Δ7 levels were significantl...
Molecular analysis of the two hESC unaffected and the two hESC SMA Type 1 lines revealed no SMN1 exp...
The universal presence of a gene (SMN2) nearly identical to the mutated SMN1 gene responsible for Sp...
<p>Representative immunoblots of both differentiated and undifferentiated mESCs revealed a significa...
<p>SMN2 mRNA was isolated from blood and analyzed using qRT-PCR. Expression level is calculated usin...
<p>The “SMN2 copy number” and “SMN-FL transcript” columns include the partial correlation analysis f...
<p>(<b>A</b>) Immunoblot of SMN and β-actin following knockdown of SRSF3 with siRNA in SMA patient-d...
<p>(<b>A</b>) Immunoblots of SMN and β-actin following knockdown of SR proteins in GM00232 type 1 SM...
(A) Multiple comparisons of SMN concentrations in untreated or ASO-treated groups with and without P...
<p>(A) Analysis of cell proliferation in wild-type (Control), NIH3T3-Smn<sub>RNAi</sub>, NIH3T3-SMN2...
<p>(A) SMN levels with respect to age in all subjects. (B) SMN protein levels were measured in SMA p...
BACKGROUND: Clinical trials to test safety and efficacy of drugs for patients with spinal muscular a...
<p>While SMN protein levels are lower in SMA relative to Control subjects, protein levels by SMA typ...
<p>SMN levels were analyzed by CD+ cell subtype at the 7d timepoint from Study 4. <b>A</b>: Analysis...
<p>A,C: SMN2-FL, SMN-FL and Total SMN transcripts generally increase with SMA Type. SMN-FL (A) is a ...
<p>(A) SMN2 mRNA expression levels in blood from patients with SMA. SMN2-Δ7 levels were significantl...
Molecular analysis of the two hESC unaffected and the two hESC SMA Type 1 lines revealed no SMN1 exp...
The universal presence of a gene (SMN2) nearly identical to the mutated SMN1 gene responsible for Sp...
<p>Representative immunoblots of both differentiated and undifferentiated mESCs revealed a significa...
<p>SMN2 mRNA was isolated from blood and analyzed using qRT-PCR. Expression level is calculated usin...
<p>The “SMN2 copy number” and “SMN-FL transcript” columns include the partial correlation analysis f...
<p>(<b>A</b>) Immunoblot of SMN and β-actin following knockdown of SRSF3 with siRNA in SMA patient-d...
<p>(<b>A</b>) Immunoblots of SMN and β-actin following knockdown of SR proteins in GM00232 type 1 SM...
(A) Multiple comparisons of SMN concentrations in untreated or ASO-treated groups with and without P...
<p>(A) Analysis of cell proliferation in wild-type (Control), NIH3T3-Smn<sub>RNAi</sub>, NIH3T3-SMN2...