BC Innovations | Oct 11, 2018
Distillery Therapeutics


INDICATION: Metabolic Mouse studies suggest base editing-mediated knockout of HPD could help treat tyrosinemia type I, which is caused by mutations in FAH downstream of HPD. In an in utero mouse model of the disease...
BC Innovations | Aug 18, 2016
Distillery Therapeutics

Therapeutics: Fumarylacetoacetate hydrolase (FAH)

Endocrine/metabolic disease INDICATION: Metabolic Mouse and pig studies suggest ex vivo FAH gene therapy could help treat hereditary tyrosinemia type 1, which is caused by FAH deficiency. In a mouse model of the disease, transplants...
BC Innovations | Jul 21, 2016
Translation in Brief

Survival of the modified

A selection strategy commonly used to clone cells in vitro has been co-opted by a group at Oregon Health & Science University to create a gene therapy platform that gives a proliferation advantage to cells...
BC Innovations | Jun 30, 2016
Distillery Techniques

Techniques: In vivo selection of genetically modified hepatocytes to improve efficacy of hepatocyte-targeting gene therapies

Drug platforms TECHNOLOGY: Gene therapy A method for selecting genetically modified hepatocytes in vivo could help improve the efficacy of gene therapies for hepatic diseases. The method involved co-administering a liver-targeted, genome-integrating vector encoding the...
BC Innovations | Oct 2, 2014
Tools & Techniques

A CRISPR possibility for DMD

CRISPR-based genome editing has filled the headlines since its discovery about two years ago for its ability to modify specific sequences in the genome with high accuracy, but its impact to date has been mainly...
BC Innovations | Apr 24, 2014
Cover Story

CRISPR in the liver

Despite the fact that the therapeutic utility of CRISPR-based approaches has yet to be demonstrated, venture dollars keep flowing into new companies developing the platform. But proof of concept may come faster than expected as...
BC Innovations | Apr 17, 2014
Distillery Techniques

Technology: Drug platforms

Approach Summary Licensing status Publication and contact information Drug platforms Clustered, regularly interspaced short palindromic repeats (CRISPR)-based genome editing platform to treat genetic liver disease Mouse studies suggest CRISPR-based genome editing could be used to...
BC Innovations | Sep 27, 2012
Distillery Techniques

Technology: Disease models

Approach Summary Licensing status Publication and contact information Disease models Humanized mouse model for liver-stage malaria infection Mice with humanized livers could be useful for identifying compounds that target the liver stage of human malarial...
BC Innovations | Sep 27, 2012
Tools & Techniques

Humanizing malaria mice

U.S. researchers have developed the first mouse model that recapitulates the human stages of the malarial parasite Plasmodium falciparum life cycle in one animal. 1 The academics plan to make additional modifications to the mice...
BC Week In Review | Nov 6, 2000
Company News

StemCells other research news

STEM researchers and colleagues published in Nature Medicine that injection of adult bone marrow cells restored liver function in a mouse model of tyrosinemia type I, which results from a deficiency in the fumarylacetoacetate hydrolase...
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