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Roussange et al., 2026 - Integrative analysis of drug-gene signatures in human pluripotent stem cells reveals prazosin as a novel SQSTM1 regulator for ALS therapeutics
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Figure 1 Molecular screening of repositioning drugs by RNA-seq identifies new therapeutic candidates for rare diseases
(A) Schematics illustrate the experimental paradigm. After 24h of treatment with drugs at 10μM, mRNAs from hES-derived MPCs were extracted and submitted to transcriptomic analysis to identify therapeutic regulation of genes involved in monogenic diseases, referenced in the OMIM database.

(B) Graphs show the number of drugs that regulate alternative splicing (LEFT) or transcript expression (RIGHT), categorized based on the number of events significantly altered compared to the control.

(C) Distribution of the different types of alternative splicing events induced by drug treatments.

(D) Graphs display the number of genes significantly regulated by each drug with log2 fold change >1 or ΔPSI >10% (blue) as well as the proportion of genes referenced in OMIM (red).

(E) Categorization of regulated genes referenced in OMIM by disease family.

(F) Normalized expressions of EGR2, GDAP1, PMP22, LMNB1, SOD1, DES, PLP1, and SQSTM1 genes analyzed by quantitative RT-PCR in hES-derived MPCs treated with different drugs at 10μM for 24h. Data are shown as mean ± SD of three independent cultures and experiments.

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