CRISPR: Implications for materials science
CRISPR: Implications for materials science The latest possibilities for editing DNA with pinpoint accuracy are transforming. CRISPR-Cas9 is a method of genome editing that exploits a natural DNA-snipping enzyme in bacteria, called Cas9 (CRISPR-associated protein 9) to target and edit particular genes. CRISPR stands for Clustered regularly interspaced short palindromic repeats, which are segments of […]
The first human trial of CRISPR-based cell therapy
The first human trial of CRISPR-based cell therapy clears safety concerns as new treatment for late-stage lung cancer In a study recently published in Nature Medicine, Lu et al. examined the feasibility and safety of using CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats)-engineered, patient-derived T cells to treat late-stage lung cancer.
Massively targeted evaluation of therapeutic CRISPR off-targets in cells
Massively targeted evaluation of therapeutic CRISPR off-targets in cells Abstract Methods for sensitive and high-throughput evaluation of CRISPR RNA-guided nucleases (RGNs) off-targets (OTs) are essential for advancing RGN-based gene therapies. Here we report SURRO-seq for simultaneously evaluating thousands of therapeutic RGN OTs in cells. SURRO-seq captures RGN-induced indels in cells by pooled lentiviral OTs libraries […]
CAD mutations and uridine-responsive epileptic encephalopathy
CAD mutations and uridine-responsive epileptic encephalopathy Unexplained global developmental delay and epilepsy in childhood pose a major socioeconomic burden. Progress in defining the molecular bases does not often translate into effective treatment. Notable exceptions include certain inborn errors of metabolism amenable to dietary intervention. CAD encodes a multifunctional enzyme involved in de novo pyrimidine biosynthesis. Alternatively, pyrimidines can be recycled […]