Microphthalmia and Disrupted Retinal Development Due to a Knock-in/Knock-Out Allele at the Locus.

Purpose: Visual System Homeobox 2 () is a transcription factor expressed in the developing retina that regulates tissue identity, growth, and fate determination. Several mutations in the gene exist in mice, including a spontaneous nonsense mutation and two targeted missense mutations originally identified in humans. Here, we expand the genetic repertoire to include a reporter allele ( ) designed to express beta-Galactosidase (bGal) and simultaneously disrupt function (knock-in/knock-out).

Microphthalmia and disrupted retinal development due to a knock-in/knock-out allele at the locus.

Visual System Homeobox 2 () is a transcription factor expressed in the developing retina that regulates tissue identity, growth, and fate determination. Several mutations in the gene exist in mice, including a spontaneous nonsense mutation and two targeted missense mutations originally identified in humans. Here, we expand the genetic repertoire to include a reporter allele ( ) designed to express beta-Galactosidase (b-GAL) and simultaneously disrupt function (knock-in/knock-out).

Porous Silicon Nanoparticles Targeted to the Extracellular Matrix for Therapeutic Protein Delivery in Traumatic Brain Injury.

Traumatic brain injury (TBI) is a major cause of disability and death among children and young adults in the United States, yet there are currently no treatments that improve the long-term brain health of patients. One promising therapeutic for TBI is brain-derived neurotrophic factor (BDNF), a protein that promotes neurogenesis and neuron survival. However, outstanding challenges to the systemic delivery of BDNF are its instability in blood, poor transport into the brain, and short half-life in circulation and brain tissue.

Pharmacokinetic Analysis of Peptide-Modified Nanoparticles with Engineered Physicochemical Properties in a Mouse Model of Traumatic Brain Injury.

Peptides are used to control the pharmacokinetic profiles of nanoparticles due to their ability to influence tissue accumulation and cellular interactions. However, beyond the study of specific peptides, there is a lack of understanding of how peptide physicochemical properties affect nanoparticle pharmacokinetics, particularly in the context of traumatic brain injury (TBI). We engineered nanoparticle surfaces with peptides that possess a range of physicochemical properties and evaluated their distribution after two routes of administration: direct injection into a healthy mouse brain and systemic delivery in a mouse model of TBI.