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Innovative gene therapy allows complete remission of clinical signs of sickle cell disease

A team led by Pr. Marina Cavazzana conducted at Necker Children's Hospital, AP-HP and the Imagine Institute in October 2014 gene therapy in the context of a phase I clinical trial / II in a patient 13 years with severe sickle cell disease.

2 Mar 2017

Scientists secure NIH grant to test portable blood-adhesion monitor for sickle cell disease patients

Researchers at Case Western Reserve University will use a $2 million grant from the National Institutes of Health to develop and test a small, portable blood-adhesion monitor for sickle cell disease patients.

8 Feb 2017

Researchers explore ins and outs of genome sequencing in newborns

When you have a baby, a nurse or a phlebotomist performs a heel stick to take a few drops of blood from your infant and sends it off to a state lab for a battery of tests.

19 Jan 2017

NIH scientists create Salmonella-infected mouse model to study life-threatening meningitis

National Institutes of Healthscientists have established in mice a way to study potentially life-threatening meningitis caused by Salmonella.

9 Dec 2016

ASGCT seeks to educate public and policy-makers on fundamentals of gene editing

On Nov. 21, 2016, the American Society of Gene & Cell Therapy released Therapeutic Gene Editing: an ASGCT White Paper, intended to provide policy-makers, patient advocates, and the interested public with the necessary background information in anticipation of an upcoming consensus report on human gene editing from the National Academy of Sciences and National Academy of Medicine (NAS), expected to be released in early 2017.

24 Nov 2016

Scientists uncover novel process by which APOL1 gene contributes to renal disease

A Children's National Health System research team has uncovered a novel process by which the gene APOL1 contributes to renal disease, according to a paper published November 18 in the Journal of the American Society of Nephrology.

18 Nov 2016

UT Physicians expands Sickle Cell Center to accommodate growing number of patients

For people living with sickle cell disease, chronic pain becomes an all too familiar part of everyday life. The UT Physicians Comprehensive Sickle Cell Center is working to help change that.

15 Nov 2016

Yale researchers use new gene editing technique to correct mutations that cause thalassemia

A Yale-led research team used a new gene editing strategy to correct mutations that cause thalassemia, a form of anemia.

26 Oct 2016

Study identifies causes and outcomes of severe lead poisoning in children

Although national and local policies have reduced the prevalence of lead poisoning in the United States, severe cases still occur.

19 Oct 2016

Genome engineering-based methods pave way for new treatment of patients with sickle cell disease

A team of physicians and laboratory scientists has taken a key step toward a cure for sickle cell disease, using CRISPR-Cas9 gene editing to fix the mutated gene responsible for the disease in stem cells from the blood of affected patients.

12 Oct 2016

African ancestry linked to increased kidney disease risk among Hispanic/Latino adults

African ancestry contributes to the risk of chronic kidney disease among some Hispanic/Latino adults, according to a study co-authored by Loyola University Chicago researchers.

5 Oct 2016

New mobile health app may help manage hydroxyurea treatments in sickle cell patients

The National Heart, Lung, and Blood Institute, part of the National Institutes of Health, has awarded a 6-year, $4.4 million grant to St. Jude Children's Research Hospital and collaborators to improve the use of prescribed medication by sickle cell patients.

21 Sep 2016

New HemaApp uses smartphone camera to screen for anemia

In the developing world, anemia -- a blood condition exacerbated by malnutrition or parasitic disease -- is a staggeringly common health problem that often goes undiagnosed.

7 Sep 2016

New drug holds potential for treating advanced mastocytosis

Most people have never heard of mastocytosis. It's a rare, sometimes deadly, immune disorder. Now new research may help those with advanced mastocytosis and possibly many more people, too.

6 Sep 2016

Scientists find new way to use CRISPR gene editing to help fix sickle cell disease

An international team of scientists led by researchers at St. Jude Children's Research Hospital has found a way to use CRISPR gene editing to help fix sickle cell disease and beta-thalassemia in blood cells isolated from patients.

16 Aug 2016

Researchers identify biophysical markers that may help improve treatments for sickle cell disease

An interdisciplinary, international group of researchers has found new biophysical markers that could help improve the understanding of treatments for sickle cell disease, a step toward developing better methods for treating the inherited blood disorder that affects an estimated 80,000 to 100,000 Americans each year.

8 Aug 2016

Sickle cell trait may not increase mortality risk

People who carry a gene for sickle cell disease do not appear to be at an increased risk of premature death, according to a study by researchers from Stanford University School of Medicine.

4 Aug 2016

ASH commits $3 million annual funding to help sustain promising blood disease research

With a $3 million annual commitment to support promising blood disease research amid limited National Institutes of Health funding, the American Society of Hematology--the world's largest professional society concerned with the causes and treatment of blood disorders--today announced the formal establishment of the ASH Bridge Grant program after an extended four-year pilot study.

22 Jul 2016

Long-term opioid therapy may not be effective to manage pain in sickle cell disease patients

In a small study looking at pain assessments in adults with sickle cell disease, researchers at Johns Hopkins says overall, those treated long-term with opioids often fared worse in measures of pain, fatigue and curtailed daily activities than those not on long-term opioids.

21 Jun 2016

New therapy may reduce stroke risk in severe sickle cell disease patients

Eleven-year-old Martin Mwita, of Omaha, has made more than 300 visits to health care facilities since he was a baby. Because of sickle cell disease, he's suffered three strokes and countless other health episodes.

26 May 2016