BioCentury | Aug 8, 2018
Distillery Therapeutics

Cancer

INDICATION: Pancreatic cancer Patient sample, cell culture and mouse studies suggest inhibiting HNF1A could help treat pancreatic ductal adenocarcinoma (PDAC). In PDAC patient samples, high tumor expression of a HNF1A-induced gene signature was associated with...
BioCentury | Jun 16, 2016
Distillery Therapeutics

Therapeutics: Forkhead box A1 (FOXA1); FOXA2; FOXA3; GATA binding protein 4 (GATA4); HNF1 homeobox A (HNF1A); hepatocyte nuclear factor 4 α (HNF4A; TCF) FOXA3,

Hepatic disease INDICATION: Liver fibrosis Mouse studies suggest in vivo reprogramming of liver myofibroblasts into hepatocyte-like cells could help treat liver fibrosis. In a mouse model of the disease, in vivo transduction of myofibroblasts with...
BioCentury | Mar 20, 2014
Cover Story

A liver divided

Three separate teams have directly converted human fibroblasts to proliferative liver cells and thus eliminated a key drawback of using induced pluripotent stem cells to treat damaged livers-the inability of the resulting differentiated cells to...
BioCentury | Aug 15, 2013
Distillery Techniques

Technology: Drug platforms

...treat liver diseases. In culture, iHSCs generated from mouse embryonic fibroblasts by expression of the HNF1 homeobox B (Hnf1b)...
BioCentury | Apr 11, 2013
Distillery Therapeutics

Indication: Cancer

...and contact information Cancer Ovarian cancer HNF1 homeobox B (HNF1B) Patient studies suggest genetic and epigenetic variation in HNF1B...
...and prognosis of ovarian cancers. In ovarian tumor samples, epigenetic DNA analysis showed that the HNF1B...
...ovarian tumors. In samples from 44,308 subjects, genetic analysis showed that the rs7405776 SNP in HNF1B...
BioCentury | Feb 28, 2013
Distillery Therapeutics

Indication: Endocrine/metabolic disease

...disease Diabetes; obesity HNF1 homeobox B (HNF1B); microRNA-802 (miR-802) Mouse and human studies suggest inhibiting miR-802 or increasing HNF1B...
...resistance and glucose tolerance compared with control LNAs. In the mouse models, vector-induced overexpression of Hnf1b...
...Next steps could include developing a screen to identify molecules that inhibit miR-802 and/or promote HNF1B...
BioCentury | Dec 15, 2011
Distillery Therapeutics

Indication: Cancer

Indication Target/marker/pathway Summary Licensing status Publication and contact information Cancer Liver cancer Hepatocyte nuclear factor 4a (HNF4A; TCF); microRNA-124 (miR-124) Mouse and patient sample studies suggest increasing signaling in the HNF4A-miR-124 pathway could help treat...
BioCentury | Jan 6, 2011
Distillery Techniques

Technology: Assays & screens

Approach Summary Licensing status Publication and contact information Assays & screens Patient-derived cell lines as an in vitro model for drug screening for genetically complex diseases Screening small molecules in patient-derived cell lines could help...
BioCentury | Jan 6, 2011
Tools & Techniques

Modeling genetic complexity in vitro

Harvard researchers believe they have developed a method for generating in vitro models of highly complex genetic diseases, which are difficult to model with standard genetic knockout techniques. 1 With proof of principle in a...
BioCentury | Dec 10, 2009
Distillery Therapeutics

Indication: Gastrointestinal disease

Indication Target/marker/pathway Summary Licensing status Publication and contact information Gastrointestinal disease Colitis Cadherin 1, type 1, E-cadherin (CDH1); cadherin 3, type 1, P-cadherin (CDH3); hepatocyte nuclear factor 4a (HNFA); laminin b1 (LAMB1) Genomewide association studies...
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