Backed by Google Ventures, ARCH Venture Partners and more, Chroma Medicine announced the closing of a $135 million Series B financing Wednesday.
A new study from Sanford Burnham Prebys has discovered a drug that can spur liver regeneration in patients with Alagille syndrome.
For the first time, research conducted by Associate Professor Duc Dong, Ph.D. has revealed that the detrimental effects of Alagille syndrome, a genetic disorder that has no cure, can be reversed using a single drug. The findings, published in the Proceedings of the National Academy of Sciences, have the potential to revolutionize the treatment approach for this rare condition, and could also shed light on more widespread diseases.
“Alagille syndrome is widely considered an incurable disease, but we believe we’re on the way to changing that,” says Dong, who is also the associate dean of admissions for Sanford Burnham Prebys’ graduate school. “We aim to advance this drug into clinical trials, and our results demonstrate its effectiveness for the first time.”
According to a recent study by researchers at Weill Cornell Medicine, a protein that prepares DNA
DNA, or deoxyribonucleic acid, is a molecule composed of two long strands of nucleotides that coil around each other to form a double helix. It is the hereditary material in humans and almost all other organisms that carries genetic instructions for development, functioning, growth, and reproduction. Nearly every cell in a person’s body has the same DNA. Most DNA is located in the cell nucleus (where it is called nuclear DNA), but a small amount of DNA can also be found in the mitochondria (where it is called mitochondrial DNA or mtDNA).
Adding evidence to the importance of early development, a new study links neutral maternal behavior toward infants with an epigenetic change in children related to stress response.
Epigenetics are molecular processes independent of DNA that influence gene behavior. In this study, researchers found that neutral or awkward behavior of mothers with their babies at 12 months correlated with an epigenetic change called methylation, or the addition of methane and carbon molecules, on a gene called NR3C1 when the children were 7 years old. This gene has been associated with regulating the body’s response to stress.
“There is evidence of a relationship between the quality of maternal-infant interaction and methylation of this gene though these are small effects in response to a relatively small variation in interaction,” said Elizabeth Holdsworth, a Washington State University biological anthropologist and lead author of the study published in the American Journal of Human Biology.
The iconic X-shaped organization of metaphase chromosomes is frequently presented in textbooks and other media. The drawings explain in captivating manner that the majority of genetic information is stored in chromosomes, which transmit it to the next generation. “These presentations suggest that the chromosome ultrastructure is well-understood. However, this is not the case,” says Dr. Veit Schubert from IPK’s chromosome structure and function research group.
Several models have been proposed to describe the higher-order structure of metaphase chromosomes based on data obtained using a range of molecular and microscopy methods. These models are categorized as helical and non-helical. Helical models assume that the chromatin in each sister chromatid at metaphase is arranged as a coil, whereas non-helical models suggest that chromatin is folded within the chromatids without forming a spiral.
The researchers revived the term “chromonema,” which was used for the first time at the beginning of the 20th century. Now, the IPK and IEB researchers provided a detailed description of its ultrastructure. Different experimental approaches, including chromosome conformation capture sequencing (Hi-C) of isolated mitotic chromosomes, polymer modeling, and microscopic observations of sister chromatid exchanges and oligo-FISH labeled regions at the super-resolution level provided an independent proof for the coiling of the chromonema.
Neuroscience research just got a little bit easier, thanks to the release of tens of thousands of images of fruit fly brain neurons generated by Janelia’s FlyLight Project Team.
Over eight years, the FlyLight Project Team and collaborators dissected, labeled, and imaged the neurons of more than 74,000 fruit fly brains, taken from more than 5,000 different genetically modified fly strains.
Now, these images are being made freely available, enabling scientists to quickly and easily find the neurons they need to test theories about how the nervous system works.
Bruce Willis has FTD. I always wondered if gene therapy could help. Apparently so did Passage Bio, and they are doing clinical trials.
FTD is a disorder that affects the frontal and temporal lobes of the brain, areas that control personality, executive function, and language. FTD is a form of early onset dementia and currently has no approved disease-modifying therapies. In approximately 5–10% of individuals with FTD, the disease occurs because of mutations in the GRN gene. These mutations cause a deficiency of progranulin that helps regulate cellular processes.
Continue reading “Gene Therapy Clinical Trial for Frontotemporal Dementia Has Begun” »
Northwestern Medicine scientists have identified the cause of a genetic subtype of autism and schizophrenia that results in social deficits and seizures in mice and humans.
Scientists have discovered a key feature of this subtype is a duplicated gene that results in overactive or overexcited brain circuits. The subtype is called 16p11.2 duplication syndrome.
“We found that mice with the same genetic changes found in humans are more likely to have seizures and also have social deficits,” said lead author Marc Forrest, research assistant professor of neuroscience at Northwestern University Feinberg School of Medicine.
The authors present a new approach to create and edit custom spatiotemporal neural activity patterns in awake, behaving animals with extremely high spatial and temporal precision. They present novel opsins optimized for multiphoton optogenetics.
New insights on how subunits of the influenza virus polymerase co-evolve to ensure efficient viral RNA replication are provided by a study published October 3 in the open-access journal PLOS Pathogens by Nadia Naffakh of the Institut Pasteur, and colleagues. As the authors note, the findings could lead to novel strategies for antiviral drug development.
Because of their yearly recurrence and the occasional emergence of pandemics, influenza viruses represent a worldwide major public health threat. Enhancing fundamental knowledge about the influenza RNA–polymerase, which is an enzyme that consists of three subunits (i.e., a heterotrimer) and ensures transcription and replication of the viral genome, is essential to reach the goal of better prevention and treatment of disease.
In the new study, Naffakh and colleagues gained new insights into viral polymerase function. They showed that the polymerase subunits co-evolve to ensure not only optimal inter-subunit cooperation within the heterotrimer, but also proper levels dimerization—the process by which pairs of heterotrimers attach together—which appears to be essential for efficient viral RNA replication. The findings point to influenza polymerase dimerization as a feature that can restrict genetic reassortment, a major evolutionary mechanism in which influenza viruses swap gene segments, and could become an attractive target for antiviral drug development.
