Highly efficient CRISPR/Cas9-mediated exon skipping for recessive dystrophic epidermolysis bullosa.

Gene therapy based on the CRISPR/Cas9 system has emerged as a promising strategy for treating the monogenic fragile skin disorder recessive dystrophic epidermolysis bullosa (RDEB). With this approach problematic wounds could be grafted with gene edited, patient-specific skin equivalents. Precise gene editing using homology-directed repair (HDR) is the ultimate goal, however low efficiencies have hindered progress.

Unintended CRISPR-Cas9 editing outcomes: a review of the detection and prevalence of structural variants generated by gene-editing in human cells.

Genome editing using the clustered regularly interspaced short palindromic repeats (CRISPR) and CRISPR-associated protein (Cas) gene-editing system (CRISPR-Cas) is a valuable tool for fundamental and applied research applications. Significant improvements in editing efficacy have advanced genome editing strategies into phase 3 human clinical trials. However, recent studies suggest that our understanding of editing outcomes has lagged behind the developments made in generating the edits themselves.

Epidermolysis Bullosa: A Review of the Tissue-Engineered Skin Substitutes Used to Treat Wounds.

Skin wound healing is a crucial process for regenerating healthy skin and avoiding the undesired consequences associated with open skin wounds. For epidermolysis bullosa (EB), a debilitating group of fragile skin disorders currently without a cure, skin blistering can often be severe and heal poorly, increasing susceptibility to life-threatening complications. To prevent these, investigational therapies have been exploring the use of tissue-engineered skin substitutes (TESSs) aimed at replacing damaged skin and promoting long-term wound closure.

Multiplex assessment of serum biomarker concentrations in well-appearing children with inflicted traumatic brain injury.

Proper diagnosis of mild inflicted traumatic brain injury (ITBI) is difficult; children often present without a history of trauma and with nonspecific symptoms, such as vomiting. Previous studies suggest that biomarkers may be able to screen for brain injury in this population, but these studies focused on only a few biomarkers. We hypothesized that using multiplex bead technology we would be able to identify multiple differences in the serum biomarker profile between in children with ITBI and those without brain injury.

Serum biomarker profiles and response to neoadjuvant chemotherapy for locally advanced breast cancer.

Introduction: Neoadjuvant chemotherapy has become the standard of care for the diverse population of women diagnosed with locally advanced breast cancer. Serum biomarker levels are increasingly being investigated for their ability to predict therapy response and aid in the development of individualized treatment regimens. Multianalyte profiles may offer greater predictive power for neoadjuvant treatment response than the individual biomarkers currently in use.

Development of multimarker panel for early detection of endometrial cancer. High diagnostic power of prolactin.

Objective: Endometrial carcinoma is the most common gynecologic cancer. Although the prognosis for endometrial cancer is generally good, cancers identified at late stages are associated with high levels of morbidity and mortality. Therefore, prevention and early detection may further reduce the burden of this challenging disease.

Biological control does not imply paradox.

Is the classical predator-prey theory inherently pathological? Defenders of the theory are losing ground in the debate. We will demonstrate that detractors' main argument is based on a faulty model, and that the conceptual and predictive bases of the theory are fundamentally sound.