Study Provides Hope for Patients With Blood Disorders

Using CRISPR gene editing technology new study introduces beneficial natural mutations into blood cells to boost production of foetal haemoglobin.

New study has used CRISPR gene editing technology to introduce beneficial natural mutations into blood cells to boost production of foetal haemoglobin. The study conducted by the University of New South Wales (UNSW) in Sydney, Australia, could lead to new therapies for sickle cell anaemia and other blood disorders.

The research solves a 50-year-old mystery about how these mutations -- which are naturally carried by a small percentage of people -- operate and alter the expression of human genes.

CRISPR Gene Editing for Common Blood Disorders
CRISPR gene editing could be used to exploit naturally occurring mutations as treatments for blood diseases such as beta-thalassemia and sickle cell disease, revealed researchers.

‘CRISPR gene editing technology was used to introduce beneficial natural mutations into blood cells to boost production of foetal haemoglobin.’

The details of the study, carried out by an international team led by UNSW scientist Professor Merlin Crossley, is published in the journal Nature Genetics.

Genome editing or gene editing give scientists the ability to change an organism's DNA. These technologies allow genetic material to be added, removed or altered at particular locations in the genome.

"Our new approach can be seen as a forerunner to 'organic gene therapy' for a range of common inherited blood disorders including beta thalassemia and sickle cell anaemia," said Professor Crossley, who is also UNSW Deputy Vice-Chancellor Academic.

Color-coded Blood Stem Cells Shed New Light on Cancer, Blood Disorders
Colors in the stem cells indicates blood disorders and cancers which arise when a mutant clone of an original blood stem cell starts to dominate.

"It is organic because no new DNA is introduced into the cells. Rather, we engineer in naturally occurring, benign mutations that are known to be beneficial to people with these conditions. It should prove to be a safe and effective therapy, although more research would be needed to scale the processes up into effective treatments," he added.

People with thalassemia or sickle-cell anaemia have defective adult haemoglobin -- the vital molecule that picks up oxygen in the lungs and transports it around the body -- and require life-long treatment with blood transfusions and medications.

CRISPR Gene Editing Identifies Novel Therapeutic Approach for Blood Disorders
A new way to use CRISPR gene editing tool to fix sickle cell disease and beta-thalassemia has been developed by St. Jude Children’s Research Hospital.

According to UNSW, it has engaged in a series of initiatives with the Indian government, higher education institutions, and corporations for sharing and transfer of its vast pool of tech expertise. This sets UNSW apart from host of other institutions that see India as a one-way street to train Indian students.

UNSW is home to more than 52,000 students from nearly 130 countries.

Zebrafish Hematopoiesis Offers Insights into Blood Disorders
Unique attributes of zebrafish make it useful as a model system for performing large-scale forward genetic screens.

Source-IANS