Personalized antisense oligonucleotide therapy for rare pediatric genetic disease: SCN2A
Therapeutic Candidate or Device Investigational personalized antisense oligonucleotide drug (nL-SCN2A-002) Indication SCN2a-associated genetic disorder Therapeutic Mechanism The study participant has a pathogenic de novo p.R853Q gain-of-function amino acid substitution mutation…
Embryonic stem cell-based generation of rat models for assessing human cellular therapies
Heart failure, diabetes and neurodegenerative diseases are among the leading causes of death and disability worldwide. These diseases are characterized by the loss of specific cell types and can be…
A small molecule tool for reducing the malignant potential in reprogramming human iPSCs and ESCs
This research project aims to solve a key bottleneck in the use of differentiated human embryonic stem cells and induced pluripotent stem cells for the regeneration and replacement of diseased…
Biophysical Determinants of Early Embryonic Stem Cell Fate Specification
Regenerative therapies require effective differentiation of stem cells to cell types that are functionally identical to those found in vivo. Many current differentiation protocols merely involve optimization of proteins added…
Single Molecule Biophysics and Biology of Cellular Identity
One of your earliest childhood biology lessons probably occurred when your body demonstrated to you that your skin is an organ that is able to self-regenerate. Indeed wound healing is…
hPSC-derived enteric ganglioids for cell therapy in gastrointestinal motility disorders
Research Objective The proposed aims will enable the generation, purification and characterization of enteric neurons from diverse hiPSCs and assessment of their efficacy for cell therapy in GI motility disorders.…
