Cell Biology Disease Doctors Treatment 
Drug Discovery World
OCTOBER 18, 2023
Adeno-associated virus (AAV) vector-based gene therapies hold exceptional promise across a range of disease areas. We’re beginning to see their potential come to fruition, with the FDA having approved three treatments as of January 2023 — for retinal dystrophy 1 spinal muscular atrophy, 2 and haemophilia B.
Drug Target Review
JANUARY 30, 2024
Adenylyl cyclase superactivation induced by long-term treatment with opioid agonist is dependent on receptor localized within lipid rafts and is independent of receptor internalization. Mechanisms of rapid opioid receptor desensitization, resensitization and tolerance in brain neurons. Br J Pharmacol. 2012;165(6):1704-16. Mol Pharmacol.
This site is protected by reCAPTCHA and the Google Privacy Policy and Terms of Service apply.
KIF1A
MAY 24, 2023
In this blog post, KIF1A.ORG Chief Science Officer Dr. Dominique Lessard describes a unique cross disciplinary experience with multiple scientific stakeholders in the rare disease space, all focused around our holistic understanding of KAND. How do we move the needle to make patient voices and experiences the center of scientific discovery?
Drug Discovery World
APRIL 14, 2023
He is being recognised for his revolutionary contributions to developing the first gene-edited cell-based therapy for cancer that involves the genetic re-engineering of a patient’s own T cells to combat their disease, and for demonstrating that adoptive T-cell therapy can induce remission and in some cases cure patients with advanced cancer.
Broad Institute
OCTOBER 11, 2023
By Allessandra DiCorato October 11, 2023 In 2011, Robert Manguso was working in a cell biology lab when his mother was diagnosed with Merkel cell carcinoma, a rare and aggressive skin cancer. His mother had a presentation of the disease that suggested her immune system was already on the job.
Codon
APRIL 9, 2024
“Cumulatively the deadliest of the human infectious diseases,” malaria has been documented throughout recorded history. In response to the precipitous rise in incidence, governments on both sides of the Pacific began a systematized search for antimalarial treatments. Subscribe to receive essays from Asimov Press.
Drug Target Review
JUNE 29, 2023
Success in the haematology space has really been driving this from the beginning, which is not surprising as haem doctors understand stem cell transplants; they’re used to working with a lot of the sort of toxicities that can come with cell therapies.
Expert insights. Personalized for you.
162 
Let's personalize your content