Spinal muscular atrophy (SMA) is a severe neurological disease caused by mutations in the SMN1 gene, characterized by early onset and degeneration of lower motor neurons.Understanding early neurodevel

NIAID Data Ecosystem30

Whole Genome Sequencing of Two Family Trios with 22q.11.2 Deletion Syndrome

22q11.2缺失综合征

遗传修饰因子识别

Two subjects with 22q11.2 deletion syndromes (22q11DS) and their parents were recruited for a whole genome sequencing study to identify candidate genetic modifiers of the various phenotypes seen in 22

NIAID Data Ecosystem30

Vivo-Morpholino sequences used in the present study.

反义寡核苷酸

基因功能验证

References listed indicate studies that have confirmed the effectiveness of these MO sequences. Note that no p58b or p16 sequences have ever been tested with Vivo-Morpholinos.Vivo-Morpholino sequences

Figshare2015-12-02 更新40

Transcriptional profiling of liver samples from Lmna Gly609Gly knock-in mice

早衰症模型研究

反义寡核苷酸

Hutchinson-Gilford Progeria Syndrome (HGPS) is caused by a point mutation in the LMNA gene that activates a cryptic donor splice site and yields a truncated form of prelamin A called progerin. Small a

NIAID Data Ecosystem30

An antisense oligonucleotide-based strategy to ameliorate cognitive dysfunction in the 22q11.2 Deletion Syndrome

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