SFARI | Topic: Molecular Mechanisms

Stem cell models to assess antisense oligonucleotides as novel therapeutics for autism

Even with tremendous advances in understanding of the causes, autism remains mostly without specific treatments. Antisense oligonucleotides (ASOs) are programmable drugs that can target specific mutations. Joseph Gleeson’s lab plans to generate ASOs to target specific ASD-linked mutations and assess these drugs in stem cell models for individualized therapy.

Association between resting-state functional brain connectivity and gene expression is altered in autism spectrum disorder.

Human iPSC-derived neuron of 16p11.2 deletion reveals haplotype-specific expression of MAPK3 and its contribution to variable NDD phenotypes.

FMRP-dependent production of large dosage-sensitive proteins is highly conserved.

Opposite expression patterns of Spry3 and p75NTR in cerebellar vermis suggest a male-specific mechanism of autism pathogenesis.

Transcriptional vulnerabilities of striatal neurons in human and rodent models of Huntington’s disease.

Cnih3 deletion dysregulates dorsal hippocampal transcription across the estrous cycle.

Combined omic analyses reveal novel loss-of-function NLGN3 variants in GnRH deficiency and autism.

The primary ciliary deficits in cerebellar Bergmann glia of the mouse model of fragile X syndrome.

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