Volume 25 Issue 2, February 2019

Careful sorting of probiotics may finally pave the way for an FDA approval.

Kjersti Aagaard is a practicing maternal–fetal medicine obstetrician at Texas Children’s and Ben Taub Hospitals and a reproductive biologist at Baylor College of Medicine. She studies a myriad of aspects of the microbiome, including how it can influence pregnancy and the developing infant.

Eran Elinav is an immunologist at the Weizmann Institute of Science in Rehovot, Israel. He has earned many distinctions, including being named a Howard Hughes Medical Institute and Bill and Melinda Gates Foundation research scholar in 2017.

Counteracting splice defects in the CEP290 gene using RNA antisense oligonucleotides or Cas9-mediated gene editing is a therapeutic strategy for Leber congenital amaurosis type 10—a severe untreatable retinal dystrophy leading to childhood blindness.

Vaccination with the tuberculosis (TB) vaccine Bacillus Calmette–Guérin (BCG) into the lungs of Rhesus macaques induces specific, local immune responses that delay infection in some animals and completely prevent it in others while protecting against TB disease.

Two new biomarkers for Alzheimer’s disease include one in the blood that relates to neurodegeneration and another that reflects blood–brain barrier dysfunction and is identifiable in cerebrospinal fluid analysis.

Antitumor immunity can be enhanced by blocking NKG2A, an inhibitory receptor expressed on natural killer and T cells.

The emerging field of precision epidemiology allows the personalized diagnosis, tracking and treatment of infectious diseases.

The universal flu vaccine remains elusive, but there are several strategies that scientists can take to develop one, including closer monitoring of viral evolution.

Mutational signatures in melanoma are associated with prognostic features in patients and suggest distinct disease etiologies associated with the influence of different wavelengths of ultraviolet radiation.

RNA antisense oligonucleotide therapy to restore normal splicing of a ciliopathy gene shows promising safety and efficacy results in a clinical trial to treat a form of childhood blindness.

In human cells, a humanized mouse model and non-human primates, CRISPR/Cas9 corrects the splicing defect in a gene associated with congenital blindness.

In a phase 1/2 clinical trial, gene therapy with autologous hematopoietic stem cells significantly reduced transfusion requirement in adults and children with transfusion dependent ß-thalassemia.

T cells that react to SpCas9 are observed in the peripheral blood mononuclear cells isolated from healthy humans. These findings may have implications for CRISPR–Cas9 therapeutics.

Cas9-specific antibodies and reactive T cells are found in the majority of healthy adult human serum samples analyzed. Such preexisting adaptive immunity should be taken into consideration as the CRISPR–Cas9 system moves toward clinical trials.

Delivery of the Bacillus Calmette–Guérin vaccine into the lungs but not the skin of rhesus macaques protects animals from infection with Mycobacterium tuberculosis, suggesting that immune responses elicited locally may be required for vaccine efficacy.

Fast scalable 3D bioprinting generates biocompatible and biomimetic scaffolds to precisely fit the geometries of spinal cord lesions, promote axonal regeneration, and support stem cell grafts to promote recovery from spinal cord injury in rodents.

Neuroimaging and cerebrospinal fluid analyses in humans reveal that loss of blood–brain barrier integrity and brain capillary pericyte damage are early biomarkers of cognitive impairment that occur independently of changes in amyloid-β and tau.

In a longitudinal cohort of familial Alzheimer’s disease patients, the rate of change of blood biomarker levels identifies disease carriers much earlier than absolute levels and predicts both neurodegeneration and cognitive decline.

The new RAF inhibitor PLX8394 selectively blocks ERK signaling in tumors driven by class 1 and/or class 2 BRAF mutations and BRAF fusions while maintaining a broad therapeutic window.

A combination of genome-scale CRISPR-Cas9 screening and focused small-molecule sensitivity profiling enables the discovery of therapeutically targetable tumor dependencies in rare tumors.

Oncogene cooperation enhances PD-L1 translation to promote immune evasion and metastasis in a novel model of liver cancer.

B cells facilitate breast cancer metastasis to lymph nodes through production of antibodies targeting a protein on the surface of cancer cells that stimulates tumor dissemination.

A disturbed microbial network characterizes relapsing refractory Crohn’s disease and antedates disease recurrence after surgical removal of the active disease segment.

Activating cannabinoid receptors in a newly identified neural circuit ameliorates depressive-like behaviors in mice.