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Zebrafish study supports accuracy of CRISPR-Cas9 gene editing tool

Along with the promise that CRISPR-Cas9 gene editing technology can offer new human therapies is the need to ensure its safety. A recent study showed that CRISPR-Cas9 did not produce off-target gene mutations in zebrafish. These results, published in Frontiers in Genetics, confirm previous data in animal models that the risk to the rest of the genome from gene editing is minimal.

3 Dec 2019

NIH Statement for World AIDS Day outlines efforts to end the HIV epidemic

With World AIDS Day on 1st December, we are reminded by the NIH of the work that needs to be done to end the HIV Epidemic.

28 Nov 2019

Mystery surrounds gene edited babies one year on

A year back Chinese scientist He Jiankui was in the news for all the wrong reasons. He had helped create the world’s first genetically edited human babies. After a year the world is still in dark about the whereabouts and health of the babies he had created and he himself has been not seen since January this year.

27 Nov 2019

Carbon monoxide has a beneficial side at low concentrations

Most people think of carbon monoxide as harmful, and with good reason -- the colorless, odorless gas sends 50,000 people in the U.S. to hospitals each year when their furnaces malfunction or car engines run in poorly ventilated spaces. But at low concentrations, carbon monoxide has a beneficial side that scientists are trying to harness to treat diseases, according to an article in Chemical & Engineering News, the weekly newsmagazine of the American Chemical Society.

20 Nov 2019

Pioneering CRISPR therapy leaves two patients free of blood disease symptoms

The first two clinical trials for CRISPR-based treatments for inherited blood disorders have been successful.

20 Nov 2019

Novartis announces strategy to broaden patient reach and availability of medicines in sub-Saharan Africa

Novartis announced today a new strategy to broaden patient reach and availability of its portfolio of medicines in sub-Saharan Africa, which is home to the largest underserved patient population in the world.

13 Nov 2019

Improved gene editing technique can successfully cut out undesirable genetic information

City of Hope researchers may have found a way to sharpen the fastest, cheapest and most accurate gene editing technique, CRISPR-Cas9, so that it can more successfully cut out undesirable genetic information.

7 Nov 2019

Mentorship in medical school linked to increased likelihood of fellows specializing in hematology

A survey of U.S. hematology-oncology fellows suggests medical school plays an important role in shaping their interest in pursuing careers in hematology, particularly when students are exposed to hematology and oncology as part of core clerkships in internal medicine and pediatrics.

31 Oct 2019

New collaboration aims to develop gene-based cures for sickle cell disease and HIV

The National Institutes of Health plans to invest at least $100 million over the next four years toward an audacious goal: develop affordable, gene-based cures for sickle cell disease and HIV.

23 Oct 2019

Study provides insight into feasibility of improving quality of care for SCD patients

Individuals with sickle cell disease (SCD) face the burdens of chronic illness and often racial disparities, both of which may increase vulnerability to adverse social determinants of health (SDoH). For children with SCD, living in poverty is associated with lower quality of life, higher healthcare utilization and higher complication rates.

9 Oct 2019

Medicaid expenditures for pediatric population decreased in Illinois

Researchers from the University of Illinois at Chicago are reporting in JAMA Network Open that Medicaid expenditures for children and young adults have decreased in Illinois. However, a care coordination demonstration project did not further reduce the cost of care for kids participating in the program within its first year.

4 Oct 2019

Viagra enables rapid and efficient hematopoietic stem cell mobilization in mice

The combination of two clinically approved drugs--Viagra and Plerixafor--rapidly and efficiently mobilizes blood stem cells from the bone marrow into the bloodstream in mice, researchers report October 10th in the journal Stem Cell Reports.

4 Oct 2019

Novel viral vector improves gene therapy for sickle cell disease

Researchers at the National Institutes of Health (NIH) have developed a unique, improved lentiviral vector for use in gene therapy for sickle cell disease.

3 Oct 2019

Researchers make breakthrough in gene therapy for sickle cell disease

Researchers at the National Institutes of Health have developed a new and improved viral vector--a virus-based vehicle that delivers therapeutic genes--for use in gene therapy for sickle cell disease.

2 Oct 2019

Findings could open up new research avenues into how heme tolerance occurs in humans

Most people have some idea of how important iron is to their health, but Iqbal Hamza, professor in Animal and Avian Sciences, has made his seventeen-year career at the University of Maryland all about the study of iron and heme trafficking and regulation in the body.

1 Oct 2019

Uncovering new treatment targets for pulmonary inflammation in sickle cell disease

A new study by De, Agrawal, Morrone et al, challenges the common notion that airway inflammation in Sickle Cell Disease (SCD) is secondary to asthma, even though the two disorders often coexist.

30 Sep 2019

CDC to fund sickle cell disease surveillance

The Centers for Disease Control and Prevention (CDC) has announced a funding of $1.2 million to different states in order to collect data and information from people suffering from sickle cell disease. The program called the Sickle Cell Data Collection program or SCDC is at present functional in two states - California and Georgia. With the new funds coming in, nine states would be able to collect and send population based information on the health of persons living with sickle cell disease.

27 Sep 2019

Researcher aims to make CRISPR-Cas9 system safer and more precise

The mere mention of gene editing and DNA altering might conjure up images of designer babies, but that is not what researcher Jin Liu has in mind.

26 Sep 2019

New NIH program aims to improve lives for people with inherited blood and immune diseases

People with inherited diseases of the blood and immune system can now receive treatment at the National Institutes of Health Clinical Center through a new, streamlined program that integrates expertise from many medical specialties and related basic science disciplines.