Gene Editing

What is CRISPR/Cas9?

crystal_structure_of_crispr-cas 9_in_complex_with_guide_rna_and_target_dna

The CRISPR/Cas9 system is a unique tool that allows scientists to make precise, targeted changes to the genome by deleting, replacing or inserting parts to specific locations in the DNA sequence.

CRISPR/Cas9 cuts time in half for genome sequencing and is more effective for researchers analyzing genomes, RNA, and proteins of various diseases.  CRISPR/Cas9 certainly offers great hope for the rare-disease patient community, especially as it relates to future immunotherapy breakthroughs for LMS.

How It Works:

What is CRISPR/Cas9 gene editing? Video from CNN.

CRISPR Trial for Cancer Patients Proposed

“US researchers could become the first outside China to use the gene-editing technique in the clinic.
By Katarina Zimmer | January 19, 2018ed plans for a clinical trial, which will combat cancer using human immune cells reprogrammed with CRISPR, on this week (January 15), MIT Technology Review reports. This could become the first clinical use of CRISPR outside China, where researchers became the first to use the gene-editing technique to treat patients with aggressive lung cancer in 2016.
In the new trial, T cells of 18 cancer patients will be removed, genetically modified to attack tumor cells, and reinfused into the bloodstream. One of the modifications involves extracting the gene encoding the PD-1 protein, a cell surface receptor expressed on T cells that normally keeps them from attacking other cells in the body. The other edit adds a receptor that aids T-cells in finding certain types of tumors, according to MIT Technology Review.
The Penn physicians plan to target three different types of cancer: multiple myeloma, sarcoma, and melanoma. The trial was approved by an advisory group to the National Institutes of Health in 2016, but it is not yet known when it will take place.
“We are in the final steps of preparing for the trial, but cannot provide a specific projected start date,” a Penn Medicine spokesperson tells MIT Technology Review.
The trial is financially supported by the Parker Institute for Cancer Immunotherapy, a nonprofit organization founded by billionaire Sean Parker.
Switzerland-based CRISPR Therapeutics will launch its own trial later this year for a combined CRISPR and stem cell therapy to treat β thalassemia, a type of blood disorder.”

See “CRISPR to Debut in Clinical Trials