Volume 25 Issue 6, June 2019

Companies are marrying gene editing with stem cell therapies in new clinical trials.

Karishma Kaushik’s research at University of Pune focuses on chronic wound infections, from probing the complex wound infection microenvironment to enabling personalized therapeutic approaches. She is a recipient of the Ramalingaswami Re-entry Fellowship, a program funded by the Government of India to support the research of early-career scientists and their return to the country from abroad.

Takanori Takebe is an assistant professor at the Cincinnati Children’s Hospital Medical Center and a professor at Tokyo Medical and Dental University, Japan. His research aims to develop mini organ technologies derived from human stem cells and use those in patients with rare congenital metabolic disorders.

Vijay Sankaran is a practicing pediatric hematologist and researcher at Boston Children’s Hospital, the Dana-Farber Cancer Institute and the Broad Institute of MIT and Harvard. His laboratory uses insights from human genetics to study blood cell production in health and disease.

The human microbiome has captured the attention of both researchers and the lay press. Given its emerging role in health and disease, it is imperative that studies are communicated well so that in turn the public does not misinterpret the findings.

A crucial consideration for the clinical application of any burgeoning science is when the understanding of that field can be implemented without the risk of unforeseen harm. In our opinion, the need for caution is particularly urgent with respect to increasingly prevalent applications of microbiome science to medicine.

Assessment of more than 400,000 people over the age of 40 demonstrates that homozygosity for a CCR5 variant that prevents HIV-1 infection comes at the cost of increased rates of mortality.

The combination of BRAF and MEK inhibition and anti-PD-1 is tolerable and has promising efficacy, which warrants further investigation into its use as melanoma therapy.

The diversity of the vaginal microbiome is linked to ethnicity, preterm birth and transitions to a healthier profile upon pregnancy.

Using the principles of community ecology in microbiome research will help to interpret these dynamic ecosystems and their relevance to health and disease.

Germline gene therapy has the potential to prevent inheritance of monogenic disorders but must first be optimized for accuracy.

Advances in cellular regeneration and technical approaches to spinal cord repair are on the verge of being translated into clinical trials.

Individuals homozygous for the CCR5-∆32 allele have a 21% increase in mortality rate in the UK Biobank cohort. In light of the CRISPR-baby scandal, this work highlights the need for understanding the unintended consequences of introducing mutations in humans.

A diagnostic tool based on blood RNA-seq is shown to identify causal genes and variants linked to clinical phenotypes in individuals with rare diseases for which whole-exome genetic sequencing was uninformative.

A pick-the-winner clinical trial design in patients with metastatic triple-negative breast cancer shows that immune induction with doxorubicin or cisplatin may improve clinical responses to PD-1 blockade and induce a more favorable tumor microenvironment.

Treatment with BRAF and/or MEK inhibitors followed by addition of anti-PD-L1 in BRAF-mutant melanoma patients is safe and shows promising anti-tumor activity.

Triple therapy combining BRAF and MEK inhibitors with immune checkpoint blockade may benefit a subset of patients with BRAF ^V600-mutated metastatic melanoma.

A randomized phase 2 trial testing triple combination of BRAF, MEK and PD-1 inhibition as first-line therapy in patients with BRAF-mutant melanoma shows durable responses and encouraging progression-free survival.

A new anti-CD19 CAR T cell therapy induces potent antitumor responses without causing severe cytokine-release syndrome or neurotoxicity in patients with lymphoma.

A convolutional neural network performs automated prediction of malignancy risk of pulmonary nodules in chest CT scan volumes and improves accuracy of lung cancer screening.

Study of influenza virus transmission in humans provides evidence that hemagglutinin stalk-specific antibodies correlate with protection from infection.

Colorectal cancer stages are associated with distinct microbial and metabolomic profiles that could shed light on cancer progression.

New immune biomarkers of exposure to tuberculosis may require a rethink of evidence of Mycobacterium tuberculosis infection and control.

The detrimental effects of aged blood on cognition and nervous system function in mice can be combatted by targeting brain endothelial cell dysfunction via inhibition of aberrant VCAM1 signaling at the blood–brain barrier.

Ancestry and socioeconomic factors influence predictable changes in the vaginal microbiome that occur early in pregnancy in women who experience normal term birth.

As part of the second phase of Human Microbiome Project, the Multi-Omic Microbiome Study: Pregnancy Initiative presents a community resource to help better understand how microbiome and host profiles change throughout pregnancy as well as to identify new opportunities for assessment of the risk of preterm birth.