Translational Candidate Ceviginate is a neuromimetic encapsulated in a hydrogel Area of Impact Dry mouth as a result of injury to the salivary glands by radiation therapy for head and…
Translational Candidate Autologous human CD34+ HSPC of patients with Friedreich’s ataxia, modified ex vivo using CRISPR/Cas9 to remove the GAA expansion mutation in frataxin Area of Impact Friedreich’s ataxia (FRDA)…
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…
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…
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…
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…
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…
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