Researchers have developed a method to detect and measure large unintended DNA modifications created by CRISPR-Cas9 gene-editing methods currently being investigated as treatments for sickle cell disease (SCD). High levels of unintended gene modifications that occurred at selected target sites demonstrate the need for a more careful evaluation of…
News
The risk of death from end-stage kidney disease (ESKD) has generally declined in the U.S. over the last 20 years — but not as much for people with sickle cell disease (SCD). Patients with the genetic disorder still have a higher risk of mortality than those without SCD,…
Television host KiKi Shepard’s 4th Annual Celebrity Golf Classic, taking place Monday, Nov. 14, aims to raise awareness of sickle cell disease (SCD) — and funding for programs and services at the nonprofit she founded, called the KIS Foundation. The golfing event, organized by Shepard and select corporate sponsors,…
A gene editing platform developed by Cellectis called TALEN was able to restore healthy hemoglobin production in preclinical models of sickle cell disease (SCD). That’s according to data presented by Arianna Moiani, PhD, senior scientist and team leader of Innovation Gene Therapy at Cellectis, at the 29th Congress of…
PerkinElmer has donated to an initiative that would extend newborn screening for sickle cell disease (SCD) in Ghana. The goal, which is expected to be achieved by 2030, is to boost screening from its current 4% to 50% of all babies born in the West African country. The…
Activation of a protein critical for the body’s response to low oxygen leads to increased levels of fetal hemoglobin in adults — a finding that could offer new therapeutic avenues for people with sickle cell disease (SCD) or beta-thalassemia, a study says. In cell cultures of red blood cells…
Blood flow and oxygen extraction fraction — a measure of how well oxygen is used by tissues — in the brain was improved among children with sickle cell disease (SCD) who underwent a hematopoietic stem cell transplant (HSCT), a small study showed. According to researchers, these findings may…
In a $5.4 billion deal, Pfizer fully acquired Global Blood Therapeutics (GBT), bolstering its decades-long standing in the field of rare blood disorders, particularly regarding sickle cell disease (SCD). With the newly completed acquisition — plans were announced in August — Pfizer gains GBT’s therapy portfolio and pipeline.
The Sickle Cell Disease Association of America (SCDAA), in collaboration with Forma Therapeutics, is launching a campaign to raise awareness and boost knowledge about sickle cell disease (SCD). The campaign, called “Believe it!”, will be promoted on television and radio channels, encouraging people to go through the website…
Through a collaboration with Emmaus Life Sciences, Kier “Junior” Spates — of the nationally syndicated radio program “The Steve Harvey Morning Show” — will share his experiences with sickle cell disease (SCD) and the Emmaus treatment Endari (L-glutamine). The radio show features host Harvey and a team that…
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