Intellect Neurosciences Files Orphan Drug Application in the United States for Its Clinical Candidate OX1 for the Treatment of Friedreich's Ataxia

NEW YORK, April 12, 2011 (GLOBE NEWSWIRE) -- Intellect Neurosciences, Inc. announced today that it has filed an orphan drug application with the Office of Orphan Products Development of the United States Food and Drug Administration (FDA) to have Orphan Drug Designation granted to its clinical stage drug candidate, OX1 (OXIGON™) for the treatment of Friedreich's Ataxia ("FA").

Mitochondria as a Therapeutic Target for Aging and Neurodegenerative Diseases

Curr Alzheimer Res. 2011 Apr 6.

Reddy PH, Reddy TP.
Neurogenetics Laboratory, Division of Neuroscience, Oregon National Primate Research Center, Oregon Health & Science University

Keywors: Mitochondria, postmortem brain studies, clinical studies, aging, neurodegenerative diseases, phosphorylation, mitochondrial DNA defects, mitochondrial fragmentation, decreased mitochondrial fusion.

New Method Delivers Drug to the Brain

Deliver complex drug into the the CNS is a challenge, the CNS is strongly protected by the BBB, and many substances with therapeutic high potential, frataxin, or even gene therapy, are not useful if they can not reach their therapeutic targets. It is very important to have methods to achieve this goal.

New Method Delivers Alzheimer’s Drug to the Brain
ScienceDaily (Apr. 5, 2011) — Oxford University scientists have developed a new method for delivering complex drugs directly to the brain, a necessary step for treating diseases like Alzheimer's, Parkinson's, Motor Neuron Disease and Muscular Dystrophy. read more...

Original paper: Delivery of siRNA to the mouse brain by systemic injection of targeted exosomes., Nature Biotechnology, 2011; DOI: 10.1038/nbt.1807
Lydia Alvarez-Erviti, Yiqi Seow, HaiFang Yin, Corinne Betts, Samira Lakhal & Matthew J A Wood.
Department of Physiology, Anatomy and Genetics, University of Oxford, Oxford, UK.

Mitochondria regulate autophagy by conserved signalling pathways

The EMBO Journal advance online publication 5 April 2011; doi:10.1038/emboj.2011.104

Martin Graef and Jodi Nunnari,
Department of Molecular and Cellular Biology, Davis University of California, USA

Keywords: autophagy regulation, mitochondria, protein kinase A, TOR, cellular homeostasis, mitochondrial respiratory deficiency, autophagy gene induction, neurodegeneration.

Modern Surgery For Scoliosis Has Good Long-Term Outcomes

Medical News Today, Article Date: 05 Apr 2011,

Teenagers who undergo spine fusion for scoliosis using the newest surgical techniques can expect to be doing well 10 years after surgery, according to a Hospital for Special Surgery study published online ahead of print in the TK issue of the journal Spine. Read more...

Could thiazolidinediones increase the risk of heart failure in Friedreich's ataxia patients?

Movement Disorders, 26: n/a. doi: 10.1002/mds.23711

García-Giménez, J. L., Sanchis-Gomar, F. and Pallardó, F. V.

CIBERER, Biomedical Network Research Centre on Rare Diseases, Valencia, Spain
Department of Physiology, Faculty of Medicine, University of Valencia, Valencia, Spain
Fundación del Hospital Clínico Universitario de Valencia. FIHCUV-INCLIVA, Valencia, Spain

Keywords: Friedreich's ataxia; rare disease; neurological symptoms; hypertrophic cardiomyopathy; rosiglitazone; pioglitazone; heart failure; peroxisome proliferator-activated receptor-gamma coactivator-1α.

Patient's Own Cells May Hold Therapeutic Promise After Reprogramming, Gene Correction

ScienceDaily (Apr. 4, 2011) — Scientists from the Morgridge Institute for Research, the University of Wisconsin-Madison, the University of California and the WiCell Research Institute moved gene therapy one step closer to clinical reality by determining that the process of correcting a genetic defect does not substantially increase the number of potentially cancer-causing mutations in induced pluripotent stem cells.

Read more

Younger Cardiac Patients Benefit From Hypothermia Therapy

Medical News Today, 04 Apr 2011

Young adult patients with genetic heart diseases, such as hypertrophic cardiomyopathy (HCM), substantially benefitted from therapeutic hypothermia, which could further extend the role for this treatment strategy in new patient populations. read more....
(Source: Minneapolis Heart Institute Foundation)

MHC Mismatch Inhibits Neurogenesis and Neuron Maturation in Stem Cell Allografts

Chen Z, Phillips LK, Gould E, Campisi J, Lee SW, et al. 2011 MHC Mismatch Inhibits Neurogenesis and Neuron Maturation in Stem Cell Allografts. PLoS ONE 6(3): e14787. doi:10.1371/journal.pone.0014787

The role of histocompatibility and immune recognition in stem cell transplant therapy has been controversial, with many reports arguing that undifferentiated stem cells are protected from immune recognition due to the absence of major histocompatibility complex (MHC) markers. This argument is even more persuasive in transplantation into the central nervous system (CNS) where the graft rejection response is minimal.

OPEN ACCESS

FULL TEXT PDF

Efficient mining of haplotype patterns for linkage disequilibrium mapping.

J Bioinform Comput Biol. 2010, Volume: 8, Supplementary Issue 1(2010) pp. 127-146 DOI: 10.1142/S0219720010005142

Lin L, Wong L, Leong TY, Lai PS (*).
School of Computing, National University of Singapore, Singapore.
(*) Department of Paediatrics, Yong Loo Lin School of Medicine, National University of Singapore

OPEN ACCESS, full text pdf

.../...

3.2. Friedreich ataxia
Friedreich ataxia is an autosomal recessive degenerative disease that involves the
central and peripheral nervous system and the heart. The friedreich ataxia dataset
was first reported by Liu et al. for linkage disequilibrium mapping. This dataset
contains 58 disease haplotypes and 69 control haplotypes with 12 microsatellite
markers. The gene is located between the fifth and sixth markers, approximately
9.8125cM away from the leftmost marker. The total region size in this study
is 15 cM.
.../...