BioCentury | Jun 27, 2019
Product Development

Gene therapy’s next frontier lies beyond rare, monogenic diseases

...Therapeutics Inc. Voyager Therapeutics Inc. Dopa decarboxylase (DDC) (AADC) GATA binding protein 4 (GATA4) Microtubule-associated protein tau (tau) (MAPT) (FTDP-17) Myocyte enhancer factor 2C (Mef2c) Potassium...
BioCentury | Aug 1, 2018
Distillery Therapeutics

Cancer

INDICATION: Cancer; lymphoma Patient sample, cell culture and mouse studies suggest inhibiting DPY30 could help treat MYC-driven cancers and lymphomas. In tumor samples from B cell lymphoma patients, DPY30 levels were higher in MYC-driven Burkitt...
BioCentury | Mar 20, 2018
Distillery Techniques

Biomarkers

...TECHNOLOGY: Tissue markers Tumor levels of phosphorylated MEF2C could help predict responses to chemotherapy in AML...
...to chemotherapy in AML. In 47 patients treated with chemotherapy, high tumor levels of S222-phosphorylated MEF2C...
...wild-type MEF2C expression. In a xenograft mouse model of AML, tumor-specific knock-in of a phosphorylation-deficient MEF2C...
BioCentury | Dec 5, 2017
Distillery Therapeutics

Neurology

...studies suggest NMDA receptor antagonists could help treat autism caused by MEF2C haploinsufficiency. In an MEF2C-haploinsufficient...
...autism. The generic NMDA antagonist memantine is marketed to treat Alzheimer's disease. TARGET/MARKER/PATHWAY: NMDA receptor; myocyte enhancer factor 2C (MEF2C)...
BioCentury | Nov 17, 2017
Preclinical News

Memantine-based NMDA receptor antagonist could help treat autism

...A study published in Nature Communications identified myocyte enhancer factor 2C (MEF2C) mutations as a source of abnormal brain development...
...abnormal brain development and function and suggests an NMDA receptor antagonist could help treat autism. MEF2C...
...impaired neurogenesis and decreased total hippocampal neurons and dendritic complexity compared with normal MEF2C expression. MEF2C...
BioCentury | Sep 21, 2017
Tools & Techniques

On your marks

With less fanfare than the first two waves, a quiet rise in activity is driving a revival of interest in epigenetics, despite little progress in solving the fundamental challenges that have dogged the field from...
BioCentury | Jul 31, 2017
Distillery Techniques

Disease models

TECHNOLOGY: Cell models A high throughput cell co-culture system could be used to screen therapies for HBV infection. The system utilizes a microwell plate for simultaneously generating up to 96 co-cultures of pooled primary human...
BioCentury | May 31, 2017
Distillery Therapeutics

Ophthalmic disease

INDICATION: Blindness Mouse studies suggest inhibiting MEF2D could help treat light-induced retinal degeneration. In retinal tissue samples from a mouse model of the disease, the MEF2D inhibitor tool compound carnosic acid decreased cell death and...
BioCentury | Jan 27, 2017
Emerging Company Profile

Cardiac conversion

...cells. A single administration of a gene therapy delivering GATA binding protein 4 (GATA4) , myocyte enhancer factor 2C (Mef2c)...
...administered daily for two weeks following a single dose of a gene therapy delivering GATA4, Mef2c...
...with the same Wnt and TGF beta inhibitors combined with a gene therapy delivering GATA4, Mef2c...
BioCentury | Dec 13, 2016
Distillery Therapeutics

Cancer

INDICATION: Acute lymphoblastic leukemia (ALL); B cell lymphoma Patient sample, cell culture and mouse studies suggest HDAC inhibitors could help treat patients with B cell precursor ALL expressing gene fusions involving MEF2D . Levels of HDAC9...
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