Christopher W Henderson

US Patent:

20160045617, Feb 18, 2016

Filed:

Apr 10, 2014

Appl. No.:

14/783034

Inventors:

Justin C. Lee - New York NY, US
Christopher Henderson - New York NY, US

International Classification:

A61K 48/00
A61K 38/13
A61K 31/7088
A61K 38/10
A61K 31/663
A61K 45/06

Abstract:

The present invention provides, inter alia, methods and pharmaceutical compositions for treating or ameliorating an effect of proximal spinal muscular atrophy (SMA) and methods for preventing or slowing motor neuron death in a subject having SMA. The methods include administering to a subject in need thereof a modulator of a gene selected from the group consisting of phosphodiesterase 1c (Pde1c), Calbindin 2 (Calb2), Egl nine homolog 3 (Eg13), Metabotropic glutamate receptor 8 (mGluR8), Syn aptotagmin 1 (Syt1), CUGBP, Elav-like family member 4 (Celf4), and combinations thereof in an amount effective to treat or ameliorate an effect of SMA. Also provided are methods for preventing or slowing motor neuron death.

US Patent:

20150285788, Oct 8, 2015

Filed:

Oct 31, 2013

Appl. No.:

14/439620

Inventors:

Hynek WICHTERLE - New York NY, US
Christopher E. HENDERSON - New York NY, US
Sebastian THAMS - New York NY, US

International Classification:

G01N 33/50
A61K 31/407
A61K 31/404
A61K 31/519

Abstract:

The present invention provides, inter alia, methods for identifying a candidate agent that may be effective to treat or ameliorate an effect of a neurodegenerative disease in a subject. These methods include: (a) contacting a wildtype neuron and a mutant neuron with a stressor which is effective to accelerate the degeneration of the mutant neuron; (b) further contacting the wildtype neuron and the mutant neuron from step (a) with a candidate agent; and (c) determining whether the candidate agent lowers a wildtype to mutant survival ratio or increases both wildtype and mutant neuron survival.