BioCentury | Sep 26, 2018
Distillery Therapeutics


INDICATION: Leukodystrophy Cell culture, worm and mouse studies suggest inhibiting HDAC6 and other HDACs could help treat Cockayne syndrome, a leukodystrophy caused by ERCC6 mutations that is associated with defects in autophagy, neurogenesis and neuronal...
BioCentury | Jun 13, 2018
Distillery Techniques

Disease models

TECHNOLOGY: Cell models A method of directly reprogramming human adult T cells into neurons could be used to generate cell-based models of neurological diseases. The method involves transfecting primary human T cells with a combination...
BioCentury | Jun 5, 2018
Preclinical News

T cells can be converted into neurons

In a Proceedings of the National Academy of Sciences paper , a Stanford University team described a method to directly convert human blood-derived T cells into functional neurons. The new method could facilitate patient-derived cell-based studies...
BioCentury | Apr 17, 2018
Preclinical News

Salk creates brain organoids with functional blood vessels

Researchers at the Salk Institute for Biological Studies created human brain organoids that recruited and developed functional blood vessels when grafted into mice. The approach could help develop organoid models that come closer to mirroring...
BioCentury | Apr 3, 2018
Distillery Techniques

Drug platforms

TECHNOLOGY: Gene therapy Nuclear-targeted, catalytically inactive Ruminococcus flavefaciens Cas13d could be used for therapeutic targeting of single-stranded RNAs and RNA splicing in cell nuclei. The fusion consists of Cas13d from the XPD3002 strain of R....
BioCentury | Mar 29, 2018
Tools & Techniques

Cutting-edge diagnostics

Following the splash that CRISPR has made in the world of therapeutics, academics are expanding the tool kit of Cas enzymes and finding new uses in diagnostics. Early hints suggest the technology could become competitive,...
BioCentury | Mar 16, 2018
Preclinical News

New Cas enzyme can edit RNA, target tau

In Cell , researchers from the Salk Institute for Biological Studies discovered a new RNA-targeting CRISPR enzyme, CRISPR-associated protein 13d (Cas13d), and showed it could be used to edit the transcript of a frontotemporal dementia target,...
BioCentury | May 8, 2017
Distillery Techniques

Drug platforms

TECHNOLOGY: Cell therapy An in vitro differentiation protocol could generate V2a interneurons from HPSCs for use as cell therapies to treat SCI. The protocol had three steps: treating HPSCs with two tool compounds that inhibit...
BioCentury | Feb 23, 2017
Translation in Brief

Folded brain

As organoids become increasingly sophisticated tools for preclinical research, a team led by mouse genetics guru Rudolf Jaenisch has developed an organoid model of the cerebrum containing surface folding that recapitulates the brain structure in...
BioCentury | Feb 22, 2017
Distillery Techniques

Disease models

TECHNOLOGY: 3-D models Cerebral organoids derived from hPSCs could be used to model neurodevelopment diseases and screen for therapies. The organoids were generated in two stages: culturing hPSCs with fibroblast growth factor (FGF) , retinoic acid...
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