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THE BIOWORLD BIOME: Our Habitat for All Things Science


As gene editing of blood stem cells is progressing toward clinical application, one question has been how to make the technology simple and cheap enough to make it a realistic option for the large numbers of patients who could potentially benefit. (See BioWorld Today, Oct. 21, 2016.)

By combining immunotherapy targeting the innate and adaptive immune systems, researchers have been able to eliminate large, established tumors in animal models. The four-component therapeutic strategy will "certainly not [be] trivial to translate," co-corresponding author Darrell Irvine noted. But the work is proof of principle that "there is some combination treatment in immunotherapy that allows the elimination of the majority of large tumors in animal models."


HONG KONG — Scientists in Japan have identified an essential amino acid that is vital for the maintenance of hematopoietic stem cells (HSCs) in mice. That is a key finding that may lead to the development of a safer, more targeted pre-treatment regimen for bone marrow transplantation (BMT) in humans and possibly even a new approach to cancer therapy.


A new device greatly simplifies cell-based gene therapy, turning it from a clean room to a desktop operation. The device, which its developers have named "gene therapy in a box," is based on the commercially available Clinimacs Prodigy device (Miltenyi Biotec GmbH).

Using CRISPR to edit the globin gene in blood stem cells has resulted in the highest level of gene correction achieved to date in preclinical models of sickle cell disease (SCD). To be sure, "highest" is still not particularly high. The study, which was published in the Oct. 12, 2016, issue of Science Translational Medicine, showed successful gene correction in 2 percent to 5 percent of blood stem cells.


HONG KONG – A Chinese study has for the first time provided preclinical proof of concept that the targeting of tyrosine protein 6 (PTK6), also known as breast tumor kinase (BRK), with a small-molecule kinase inhibitor may represent a promising new breast cancer treatment strategy.


By studying drug mechanisms of actions in living cells, researchers have been able to show that the ocular toxicity of inhibitors of the beta-secretase beta-site APP-cleaving enzyme 1 (BACE1), which are in clinical trials for the treatment of Alzheimer's disease (AD), is due to inhibition of the related enzyme Cathepsin D.

Researchers were able to reverse memory deficits in a mouse model of Alzheimer's disease by preventing cleavage of the protein tau by the enzyme caspase-2. There's a soluble aspect of tau that disrupts synaptic function, and we have identified a key player in why that occurs – the caspase-2 cleavage of tau," said Karen Ashe.


HONG KONG – A University of Hong Kong (HKU) phase II trial has shown that a drug derived from an herb used in traditional Chinese medicine to treat acute myeloid leukemia (AML) has helped patients with an aggressive treatment-refractory form of AML to achieve remission.

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