RNA Single-base Editing Therapy that Treats Genetic Lung and Liver Disease Entered Clinical Trials
When Peggy's mom was in her forties, she started to have trouble breathing. Although she had never smoked, she had been exposed second-hand earlier in her life. Eventually, her mother ended up at the Mayo Clinic, where she was the 36th patient to be diagnosed with Alpha-1 Antitrypsin Deficiency (AATD). Knowing it was a genetic condition, the doctors tested ...
Jennifer Doudna – Seeking to Improve the World with CRISPR
At 10:53 p.m. on Oct. 7, Jennifer Doudna woke to a buzzing sound. Picking up her phone — which she noticed had multiple missed calls and messages — she was greeted by the voice of Nature journalist Heidi Ledford. The day before, Doudna had been in an all-day meeting; feeling exhausted and not thinking too much about the imminent ...
FDA Approved the First CRISPR Treatment – Potential Cure for People with Sickle Cell Disease
Victoria Gray was diagnosed with sickle cell disease (SCD) when she was just three months old; since then, episodes of pain and frequent hospitalizations have been a part of her life. Many of her dreams seemed like far-off impossibilities when the smallest things, like changing weather, would put her in the hospital. Every four to six weeks, she would ...
Follow-up Data Confirms That Inclisiran Provides Long-Term Reduction of LDL-Cholesterol
Cardiovascular disease is the leading cause of death in the United States, exceeding all types of cancer, unintentional injury, and stroke combined. However, updated data from Novartis's open-label trial shows promising long-term results for inclisiran (Leqvio), the first and only small interfering RNA (siRNA) therapy to reduce low-density lipoprotein cholesterol (LDL-C). ORION-8 is part of an extensive clinical trial ...
The First FDA Approval for a GalNAc-conjugated ASO
It starts with a pins-and-needle sensation in your feet, or maybe your gastrointestinal tract has become easily irritated, or you've started to lose weight or develop heart problems. Hereditary ATTR amyloidosis can present differently among patients and with symptoms found in far more common diseases, so it is often misdiagnosed. Fortunately, treatment for the disease is expanding, with the ...
Base Editing in Clinical Trials to Treat Acute Lymphoblastic Leukemia
Base editing has experienced a rapid rise in use since it first came on the scene in 2016, with multiple trials underway, testing its ability to treat conditions with precise, single-letter changes to DNA. The technique presents a potentially more accurate and safer method compared to nuclease-based approaches; however, it’s not without its risks. Currently, researchers are investigating how ...
RNA Single-base Editing Therapy that Treats Genetic Lung and Liver Disease Entered Clinical Trials
When Peggy's mom was in her forties, she started to have trouble breathing. Although she ...
Jennifer Doudna – Seeking to Improve the World with CRISPR
At 10:53 p.m. on Oct. 7, Jennifer Doudna woke to a buzzing sound. Picking up ...
FDA Approved the First CRISPR Treatment – Potential Cure for People with Sickle Cell Disease
Victoria Gray was diagnosed with sickle cell disease (SCD) when she was just three months ...
Follow-up Data Confirms That Inclisiran Provides Long-Term Reduction of LDL-Cholesterol
Cardiovascular disease is the leading cause of death in the United States, exceeding all types ...
The First FDA Approval for a GalNAc-conjugated ASO
It starts with a pins-and-needle sensation in your feet, or maybe your gastrointestinal tract has ...
Base Editing in Clinical Trials to Treat Acute Lymphoblastic Leukemia
Base editing has experienced a rapid rise in use since it first came on the ...
