Research Roundup: Controlling Gene Therapy and More

Gene Therapy

Every week there are numerous scientific studies published. Here’s a look at some of the more interesting ones.

Controlling the Doses of Gene Therapy

Gene therapy is relatively new, with only a few approved therapies. The techniques typically involve taking a normal gene, inserting it into a hollowed-out virus, and injecting it into the patient, where the gene produces normal proteins that are otherwise abnormal. Researchers at The Scripps Research Institute developed a molecular switch that could potentially be embedded into gene therapies that would control dosing. They published their research in the journal Nature Biotechnology.

“I think that our approach offers the only practical way at present to regulate the dose of a gene therapy in an animal or a human,” said Michael Farzan, principal investigator of the research.

The researchers demonstrated the work by incorporating the switch into a gene therapy for anemia that produces the hormone erythropoietin. The switch suppressed expression of the gene to very low levels but could then increase the gene’s expression using injected control molecules called morpholinos. Morpholinos are already approved by the FDA as safe for other applications.

Machine Learning to Interpret Gene Regulation

Although “big data” is helpful in biological systems, the data sets are so complicated that interpreting the data is still difficult and complex. Researchers at Cold Spring Harbor Laboratory designed advanced machine learning algorithms that cut through the complexity, making the data from gene regulation more easy to understand for biologists. The algorithms are a form of artificial neural network (ANN) that appears to bridge the gap between computational tools and how biologists think.

“Deep Learning” Predicts Disease-Associated Mutations

Researchers at the University of Hong Kong developed a deep learning method to predict disease-associated mutations of the metal-binding sites in a protein. It is the first time a deep learning approach has been used to predict disease-associated metal-relevant site mutations of metalloproteins. Metal ions play important roles structurally or functionally in the pathophysiology of many human biological systems, such as zinc, iron and copper. Deficiencies in these can cause severe diseases. They utilized omics data to develop a training dataset, finding that a mutation in zinc-binding sites played a major role in breast, liver, kidney, immune system and prostate diseases, while calcium- and magnesium-binding sites are linked to muscular and immune system diseases, respectively. Iron-binding site mutations are associated with metabolic diseases.

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The Science Behind Intermittent Fasting

There are, generally speaking, two types of “intermittent fasting.” One is daily time-restricted feeding, narrowing eating times to 6-8 hours per day, and 5:2 intermittent fasting, where individuals limit themselves to one moderate-sized meal two days a week. Research suggests that the reason this works is that they trigger metabolic switching, an evolutionary adaptation to periods of food scarcity. When people eat three meals a day plus snacks, the switching does not occur. The research study also found that intermittent fasting decreased blood pressure, blood lipid levels and resting heart rates. Additional studies also suggest it can improve brain health, such as learning and memory.

Many Younger Patients with Stomach Cancer Appear to Have Distinct Disease

Mayo Clinic researchers found that many people who developed stomach cancer under the age of 60 had “genetically and clinically distinct” disease from stomach cancer patients who were older. The new, early onset type of stomach cancer appears to grow and metastasize more quickly and has a worse prognosis. It is also more resistant to traditional chemotherapy. The investigators evaluated more than 75,225 cases from several databases to review stomach cancer statistics from 1973 to 2015. The average age of stomach cancer diagnosis is 68, but there appears to be growing occurrence in individuals in their 30s, 40s and 50s.

Dementia Vaccine Successful in Animal Trials

Investigators successfully tested an experimental vaccine to remove brain plaque and tau protein aggregates linked to Alzheimer’s disease in laboratory mice. The researchers from the Institute for Molecular Medicine and University of California, Irvine and Flinders University in South Australia, believe it supports clinical trials in humans, potentially in the next two years. The vaccine was a combination of two MultiTEP epitope vaccines, AV-1959R and AV-1980R, that target amyloid-beta and tau, respectively. It is formulated in AdvaxCpG, a polysaccharide adjuvant.

RNA-Targeting Approach Successfully Blocks Driver of Parkinson’s Disease

Researchers at Scripps Research in Florida developed a compound that prevents production of an underlying cause of Parkinson’s disease, an abnormal protein called alpha-synuclein. Dubbed Synucleozid, the protein halts the ribosome from detecting the messenger RNA (mRNA) template, preventing the translation of the disordered alpha-synuclein protein. This proof-of-concept study hints that the compound could become a potential Parkinson’s drug candidate.

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