Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy.

Background: Spinal muscular atrophy type 1 (SMA1) is a progressive, monogenic motor neuron disease with an onset during infancy that results in failure to achieve motor milestones and in death or the need for mechanical ventilation by 2 years of age. We studied functional replacement of the mutated gene encoding survival motor neuron 1 (SMN1) in this disease.

Methods: Fifteen patients with SMA1 received a single dose of intravenous adeno-associated virus serotype 9 carrying SMN complementary DNA encoding the missing SMN protein.

Patterns of Tamarix water use during a record drought.

During a record drought (2006) in southwest Kansas, USA, we assessed groundwater dynamics in a shallow, unconfined aquifer, along with plant water sources and physiological responses of the invasive riparian shrub Tamarix ramosissima. In early May, diel water table fluctuations indicated evapotranspirative consumption of groundwater by vegetation. During the summer drought, the water table elevation dropped past the lowest position previously recorded.