It’s an exciting time for gene-therapy and rare disease, the first in utero transplant is a success and the benefits of doing nothing.
(Genetic Engineering & Biotechnology News, 5/29/2018, Brandon May)
With our upcoming World Congress focused on rare diseases, we couldn’t resist including this one in our weekly roundup. The US National Institutes of Health has launched the Therapeutics for Rare and Neglected Diseases (TRND), a program that oversees a suite of pilot projects to address specific obstacles in gene therapy development. Rare disease research is really big right now. Gene therapy owes its new vitality to a better understanding of rare diseases, the discovery of new gene delivery vectors, and the development of site-specific genome editing technology. Already pushing up new shoots, gene therapy may flourish if it extends its roots deep into an increasingly fertile manufacturing base. Manufacturing trends that nurture gene therapy’s growth include the development of production systems for adeno-associated virus (AAV) vectors for gene delivery. All these advances are encouraging gene-therapy developers to stake new claims in rare disorder territory, which is where gene therapy was first applied, before most clinical trials turned from monogenic disorders, such as severe combined immunodeficiency, cystic fibrosis, and Duchenne muscular dystrophy (DMD), to polygenic disorders (such as heart disease, diabetes, and cancer).
(The Scientist, 5/30/2018, Jim Daley)
Pediatric surgeons at the University of California, San Francisco (UCSF, have treated a second-trimester fetus with stem cells taken from her mother’s bone marrow. The baby, born in February, was the first patient enrolled in the world’s first clinical trial using stem cells transplanted prior to birth. She is “apparently healthy,” despite living with a deadly genetic disease called alpha thalassemia, according to a statement from the university. Tippi MacKenzie, a pediatric surgeon at UCSF Benioff Children’s Hospital, led the team that performed the transplant. “It is too early to say how effective the stem cell transplantation will be, but we are encouraged by how well she and her mother have tolerated the treatment,” she says in the statement. “Her healthy birth suggests that fetal therapy is a viable option to offer to families with this diagnosis.”
(Live Science, 5/31/2018, Simon Gottschalk)
Our lives are so full of constant alerts and digital intrusion that it may seem like our head is going to explode. In a society propelled by the twin engines of acceleration and excess, doing nothing is equated with waste, laziness, lack of ambition, boredom or “down” time. But this betrays a rather instrumental grasp of human existence. Much research – and many spiritual and philosophical systems such as Buddhism suggest that detaching from daily concerns and spending time in simple reflection and contemplation are essential to health, sanity and personal growth. Similarly, to equate “doing nothing” with nonproductivity betrays a short-sighted understanding of productivity. In fact, psychological research suggests that doing nothing is essential for creativity and innovation, and a person’s seeming inactivity might actually cultivate new insights, inventions or melodies.
—Compiled by Social Media Specialist Jillian Scola