A single injection of SOD1-targeting RNA into the subpial space, which is below the innermost meningeal layer, was able to spread throughout the spinal cord and, via retrograde delivery, into brain centers that project to the spinal cord in several animal models, including primates.
ORLANDO, Fla. – Two preclinical presentations at the 61st American Society of Hematology (ASH) annual meeting could pave the way for using hematopoietic stem cell transplants (HSCT) in patients who are currently too sick to tolerate the procedure, as well as in indications where its toxicities preclude its use.
ORLANDO, Fla. – At the 61st ASH annual meeting late-breaking abstracts session, researchers from Boston Children’s Hospital reported that three adult patients who had received an autologous transplant of gene-edited hematopoietic stem cells lacking BCL11A produced high levels of functional hemoglobin and had reduced disease symptoms for at least eight months after transplantation.
ORLANDO, Fla. – Two preclinical presentations at the 61st American Society of Hematology (ASH) annual meeting could pave the way for using hematopoietic stem cell transplants (HSCT) in patients who are currently too sick to tolerate the procedure, as well as in indications where its toxicities preclude its use.
