One-Time Treatment: Base Editing Shows Promise vs. SMA
A research team led by gene editing pioneer David R. Liu, PhD, reports the application of base editing technology to develop a one-time treatment for spinal muscular atrophy (SMA), showing promising effectiveness in cell and mouse models.
The approach detailed by Liu and colleagues was one of dozens of genome editing approaches they explored for potential in treating SMA. The 79 (to be precise) approaches included either CRISPR-Cas9 nucleases or base editing strategies. All 79 targeted five regions of SMN2 to induce either post-transcriptional or post-translational regulatory changes in SMN2 that upregulate SMN protein production.
The researchers found that one-time in vivo co-administration of Spinraza and base editor extended animal lifespan nearly seven-fold, from an average of 17 days untreated to an average of 111 days, along with substantial rescue of motor function in behavioral assays such as inverted screen hang time or the time required for the mice to right themselves from being on their backs.
Spinraza won the FDA’s first approval for an SMA treatment in in 2016 and is now one of three drugs indicated for the disease. (The others are Evrysdi®, marketed by Roche and its Genentech subsidiary) and Zolgensma®, a one-time gene therapy designed to replace the SMN1 gene.
Unlike the other drugs, which carry broad SMA indications in children and adults, Zolgensma is approved for a narrower patient population of children under age two with SMA with bi-allelic mutations in SMN1. Last year, Novartis acknowledged that two patients died of acute liver failurefollowing treatment with its Zolgensma.
https://www.genengnews.com/base-editing/one-time-treatment-base-editing-shows-promise-vs-sma/
ارسال به دوستان