Everything from food aspiration to an asthma attack to heart failure can cause a patient to die from asphyxia, or lack of oxygen. For more than a decade, the Translational Research Laboratory (TRL) of Boston Children’s Hospital’s Heart Center has been pursuing a dream: tiny, oxygen-filled bubbles that can be safely injected directly into the blood, resuscitating patients who can’t breathe.
Vector consulted its many informants to find out which way the wind will blow in 2018. Here are their predictions for what to expect in genetics, stem cell research, immunology and more.
Gene-based therapies mature
We will continue to see successes in 2018 reflecting the maturation of gene therapy as a viable, generalizable platform for curing many rare diseases. Also, we will see exciting new applications of other maturing platforms, like CRISPR/Cas9 gene editing and oligonucleotide therapies for neurologic diseases, building on the success of nusinersen for spinal muscular atrophy. …
Sudden oxygen deprivation can happen for many reasons, from choking to aspiration to cardiac arrest. In these emergency situations, rapid oxygen delivery can mean the difference between life and death. But what if the person cannot breathe?
In the summer of 2012, John Kheir, MD, of the Heart Center at Boston Children’s Hospital, published a study in Science Translational Medicine describing an alternative oxygen delivery system. Kheir used tiny, gas-filled microparticles with a thin outer layer of lipids (fatty molecules) that combined to form a liquid foam-like substance. Injected into the bloodstream, the particles rapidly dissolved and delivered oxygen gas directly to the red blood cells in animal models. But the bubbles were very unstable and not suitable for clinical use. …