Gene Therapy

CRISPR in 2018: Coming to a Human Near You


Ever since scientists first used CRISPR-Cas9 to edit living human cells in 2013, they’ve been saying that the possibilities for using it to treat disease are virtually endless. Read more here.

Flux and Uncertainty in the CRISPR Patent Landscape


In May 2012, the University of California, Berkeley, filed a patent application for biochemist Jennifer Doudna and the University of Vienna’s Emmanuelle Charpentier, then of Umeå University in Sweden, based on their seminal observation that the bacterial CRISPR-Cas9 gene-editing system can be used to target different sequences of DNA by reprogramming the system’s small homing […]

Biomarker Tests for Molecularly Targeted Therapies


The Key to Unlocking Precision Medicine NEJM   An IOM committee focused on biomarkers for molecularly targeted therapies has issued recommendations about regulatory, reimbursement, and clinical practice issues, in order to guide precision medicine in advancing the care of patients with cancer and other diseases. This article was published on June 1, 2016, at Disclosure forms […]

The Lab Results Are In: Genes Might Be to Blame for Retrievers’ Obesity


  If you’ve ever had a Labrador retriever, you know about one of the breed’s notable traits: an unrelenting appetite. The dogs will devour anything from socks to rocks, and given the chance, they can be prone to eat themselves into obesity. By one estimate, nearly 60 percent of all Labradors are overweight or obese. […]

Gene drives aren’t ready for the wild, report concludes


Mosquitoes and other organisms normally have a 50 percent chance of passing along a gene to an offspring. A gene drive copies and pastes itself into chromosomes from both parents, ensuring it gets passed on more often. Mosquitoes and invasive species can rest easy — for now. Genetic technology called gene drives could wipe out […]

First Gene Therapy for Children is Approved in Europe


Radical treatment for rare ‘bubble boy’ disorder has a 100% survival rate The European Commission has given approval for British drug firm GlaxoSmithKline to produce the world’s first gene therapy treatment for children. GSK will produce Strimvelis, the first stem cell gene therapy to treat patients with severe combined immunodeficiency caused by adenosine deaminase deficiency […]

Genetically engineered immune cells are saving the lives of cancer patients


Immune Engineering – That may just be the start The doctors looking at Layla Richards saw a little girl with leukemia bubbling in her veins. She’d had bags and bags of chemotherapy and a bone marrow transplant. But the cancer still thrived. By last June, the 12-month-old was desperately ill. Her parents begged—wasn’t there anything? […]

The Gene Editors Are Only Getting Started


Rewriting the code of life has never been so easy. In 2012 scientists demonstrated a new DNA-editing technique called Crispr. Five years later it is being used to cure mice with HIV and hemophilia. Read more here.