FSHD stem cells: revolutionising researchJune 2, 2016
Show your support for FSHD Global Research Foundation:
2017 Charity of the Year in the Australian Charity awardsCharity of the year, Workplace giving, Supporters
Bill Moss AO on the Alan Jones Breakfast Show:
Chairman Bill Moss AO talks with Alan Jones as the Foundation is announced as the Charity of the Year in the Australian Charity Awards 2017FSHD Media, Bill Moss AO, Alan Jones
2017 Charity of the Year!:
CHARITY OF THE YEAR ANNOUNCED AT THE AUSTRALIAN CHARITY AWARDS 2017FSHD Global, Awards, Charity of the Year
I’m 10 years old and I want to tell you what living with FSHD is like for me.FSHD, Community Story, Inspiring
Join our team and start fundraising!Fundraising, How you can help, how to register
The power of workplace giving:
Start making a difference todayFundraising, Workplace Giving, How you can help!
World FSHD Day:
Get planning for this June 20World FSHD day, Fundraise
Bone Health in FSHD:
FSHD Global Research Grant 23 UpdateBone health, FSHD, Grant 23
Our Newest Partnership: Australian Opal Cutters & Pearl Divers:
We are a proud charity partner of Australian Opal Cutters & Peal Divers.Corporate partner, pearls
Get Positive - A piece by FSHD Patron Justin ReidLiving with FSHD
Open Gardens – Mount Wilson:
FSHD Global is proud to be hosting an open garden this April 29 & 30.open gardens, Mount Wilson, FSHD Global
FSHD Global proud to be involved in unique ethical partnership:
Cape York Native Title Holders Partner with Corporate Australia to Establish Economic Empowermentcharity, donations, business, ethical, indigenous
FSHD Educational Toolkits:
Getting the support you and your health professionals needFSHD, consensus, medical guide
Rare Disease Day:
Awareness is key.FSHD, RareDiseaseDay, fundraising, awareness
A major issue for FSHDfatigue, fshd, exercise, physiotherapy
Reading the science:
How to tell the good from the not so good.FSHD, facioscapulohumeral muscular dystrophy, publications, science
Facio-therapies announce breakthrough:
Facio-therapies recently announced a breakthrough development in FSHD drug discovery.research, science, fshd, facioscapulohumeral dystrophy, biotech
Exercise & FSHD:
The Latest Studyconsensus, FSHD, exercise
We are proud to be recognised by The Australian Charity Awards:
FSHD Global has been recognised as a Winner for Outstanding Achievement in the Australian Charity Awards 2016.charity, awards, FSHD
Short Story from Daniel Howe – Life with FSHD:
Lavender BluesFSHD, lavender,
Folo – Shopping for Love:
A new way of givingfolo, philanthropy, giving, charity, shopping
A new mechanism for FSHD:
New drug targetsFSHD, D4Z4, Dux 4, science
Seeking a Volunteer Graphic Designer:
Build your portfolio and make a difference at the same time!volunteer, graphic designer
FSHD Global publishes clinical practice considerations for FSHD:
FSHD Global publishes clinical practice considerations for FSHDscience
FSHD : It’s not all just about muscles.:
FSHD: It's not just about muscles.science, muscles, FSHD
FSHD stem cells: revolutionising research:
FSHD stem cells: revolutionising researchFSHD Global Research Foundation
CRISPR & FSHD:
Harnessing a New TechnologyScience, DNA
Altering Gene Expression:
New tools with big potentialScience DNA
Treatments for FSHD:
Small Molecules: Offering hope for people with FSHDNature chemical biology
FSHD stem cells: revolutionising research
A human cell model of facioscapulohumeral dystrophy provides valuable insight into this condition and an essential tool for drug discovery.
One of the most important tools for biomedical research is the existence of cell and animal models that closely resemble the human condition being studied. A good animal or cell model will produce results that are applicable to people and will advance the search for treatments and a cure. A poor animal model can give you misleading results wasting time and money.
So much research fails because of poor models. Unlike many human conditions FSHD does not have readily available cell and animal models. However, work performed by a group of scientists at Genea Biocells, and supported by research funding from FSHD Global, has changed this. This group has developed FSHD stem cells that are able to mimic FSHD inside a culture dish.
What is a stem cell?
A stem cell is basically a cell that can turn into multiple different types of cells. Embryonic stem cells are those cells in the early embryo which divide to create a full organism. The adult body also has stem cells such as those in bone marrow which are continuously dividing to create the multiple cells in blood.
The stem cells created by Genea are being used as a renewable source of mature FSHD skeletal muscle cells that can be studied in laboratories around the world.
Leslie Caron from Novogen was the lead author on the study she says “Our study demonstrate disease-specific phenotypes in muscle derived from both human embryonic and induced pluripotent stem cells affected with FSHD. These cells represent a scalable resource that provides many advantages over invasive patients biopsies and provides an invaluable tool for drug screening and the development of new therapies against FSHD.
This study may also provide new insight into FSHD pathology. As these cells are embryonic in nature, they allow the identification of new pathways associated with the early stages of the disease.”
The potential for these cells to help us better understand the biology of FSHD and fast-track drug discovery is certainly exciting.
The paper is published in the Journal Stem Cells Translational Medicine and is available here