Experimental Drug May Benefit Some Patients with Rare Form of ALS
When Columbia neurologist and scientist Neil Shneider speaks to his ALS patients who volunteer for experimental therapies, he’s unwaveringly honest.
‘Jumping gene’ enzyme can make big, precise changes to human DNA
Improved editing tool inserts gene-size sequences into human genome at specific locations, broadening potential treatments
New Gene Editor Enables Greater Precision
Ask scientists what gene editing tool is most needed to advance gene therapy, and they’d probably describe a system that’s now close to realization in the labs of Samuel Sternberg at Columbia University Vagelos College of Physicians and Surgeons and David Liu at the Broad Institute of MIT and Harvard.
2025 Columbia Precision Medicine Initiative Newsletter
In the enclosed newsletter, we outline two new objectives: to promote the clinical implementation of precision medicine and to build bioinformatics infrastructure to support genomic research.
Delivering Gene Therapy to the Lung Using Nanoparticles
Ever since the gene responsible for cystic fibrosis was discovered 25 years ago, scientists have envisaged gene therapies to correct the underlying genetic defect.