Gene Therapy 2.0
Ever since the death of Jesse Gelsinger, the gene therapy researches have been stagnated for over a decade. Jesse Gelsinger was the first person received a clinical trial for gene therapy. He suffered from ornithine transcarbamylase deficiency—a form of metabolic disorder—and he joined a clinical trial run by the University of Pennsylvania. On September 13th 1999, he was given an infusion of corrective OTC gene encased in a dose of attenuated cold virus, but unfortunately, he died four days later at the age of 19.
Early gene therapies failed in part because of the delivery mechanism. Gene-therapy researchers have surmounted many of those early problems by using viruses that are more efficient at transporting new genetic material into cells. It seems like 2017 will be an exciting year for the biotech industry since a new generation of gene therapy has already emerged—the CRISPR-Cas9, a genome editing tool that which cautious optimism is expected to start a new wave of faster, more accurate treatment for monogenetic diseases through DNA modification. Moreover, the first gene therapy to cure blindness from hereditary retinal dystrophies has been developed by Spark Therapeutics, and it is expected to hit the market in 2017. On October 2016, Spark Therapeutics made the announcement of 2017’s launch of SPK-RPE65. It is a gene therapy that targets the inherited mutation that causes blindness. By injecting new cells that will correct the genetic mutation retina, patients will be able to recover their vision.
The Cell Atlas
Cells are the basic building block of all organisms, and various human cells are the building blocks of all human tissues and organs. In 1665, Robert Hooke peered down his microscope at a piece of cork and discovered little boxes. The objective of modern genomics and cell biology is to construct the first comprehensive Cell Atlas, or map of human cells—a technological marvel that should comprehensively reveal what human bodies are actually made of and provide scientists a sophisticated new model of biology that could speed the search for drugs.
On October 13, 2016, an international group of scientists and renowned researchers met in London to discuss building the Human Cell Atlas. According to the conference, “A Cell Atlas would be a collection of cellular reference of static cell types…it would be an extremely valuable resource to empower the global research community to systematically study the biological changes associated with different diseases, understand where genes associated with disease are active in our bodies, analyze the molecular mechanisms that govern the production and activity of different cell type.”
In a recent research conducted by the French neuroscientist Grégoire Courtine shows that monkeys with spinal cord damage that paralyzed one leg quickly regained the ability to walk with a wireless connection from the brain to the spinal cord below the injury. In this study, the most important terminology to remember is “neural bypass”—two wireless implants worked together as a “brain-spine interface” to communicate nerve signals between the brains and spines of the monkeys. According to Courtine, he hopes that the system he and his colleagues developed could be transferred in the next 10 years to human for therapy that would aid in rehabilitation and improve recovery and quality of life.
At Case Western Reserve University, a middle-aged man who cannot move anything but his head and shoulder, agreed to try this neutral bypass implant in his brain. The implants were made of silicon, and they bristle with a hundred hair-size metal probes that can ‘listen’ as neurons fire off commands. In this experiment, this volunteer can slowly raising his arm with the help of a spring-loaded arm rest, and willing his hand to open and close. He can even raises a cu with a straw to his lips. This trial is only a part of a broader effort to use implanted electronics to restore various senses and abilities. In the future, scientists hope to use neural prosthetics to reverse blindness with chips placed in the eye, and maybe restore memories lost to Alzheimer’s disease.