A new fast-track review for UCSF gene therapy could help more kids with deadly Artemis-SCID disease get life-saving treatment sooner.
Circa 2017
Schizophrenia is a genetically related mental illness, in which the majority of genetic alterations occur in the non-coding regions of the human genome. In the past decade, a growing number of regulatory non-coding RNAs (ncRNAs) including microRNAs (miRNAs) and long non-coding RNAs (lncRNAs) have been identified to be strongly associated with schizophrenia. However, the studies of these ncRNAs in the pathophysiology of schizophrenia and the reverting of their genetic defects in restoration of the normal phenotype have been hampered by insufficient technology to manipulate these ncRNA genes effectively as well as a lack of appropriate animal models. Most recently, a revolutionary gene editing technology known as Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/CRISPR-associated nuclease 9 (Cas9; CRISPR/Cas9) has been developed that enable researchers to overcome these challenges. In this review article, we mainly focus on the schizophrenia-related ncRNAs and the use of CRISPR/Cas9-mediated editing on the non-coding regions of the genomic DNA in proving causal relationship between the genetic defects and the pathophysiology of schizophrenia. We subsequently discuss the potential of translating this advanced technology into a clinical therapy for schizophrenia, although the CRISPR/Cas9 technology is currently still in its infancy and immature to put into use in the treatment of diseases. Furthermore, we suggest strategies to accelerate the pace from the bench to the bedside. This review describes the application of the powerful and feasible CRISPR/Cas9 technology to manipulate schizophrenia-associated ncRNA genes. This technology could help researchers tackle this complex health problem and perhaps other genetically related mental disorders due to the overlapping genetic alterations of schizophrenia with other mental illnesses.
Keywords: CRISPR/Cas9; gene editing; lncRNAs; miRNAs; non-coding RNAs; schizophrenia.
Circa 2020
A startup has built what it claims is the “world’s first true smart contact lens” with an embedded display that would bring augmented reality experiences closer to your eyeball than ever before.
The company is called Mojo Vision, and its Mojo Lens is the culmination of over a decade of research, development, and patent filings (it’s racked up over 100 patents to date). While it’s not shipping a product (yet), the company is currently demonstrating a working prototype.
Continue reading “These ‘smart’ contact lenses have a built-in display for augmented reality” »
For instance, when training a gestational age clock model from placental methylation, a sample can only be collected after delivery of the baby and the placenta. So most samples have a gestational age greater than 30 weeks, which corresponds to moderate preterm and full-term births. For samples with a further younger gestational age, they are scarce, which makes the sample distribution seriously biased to large gestational ages and impairs the ability of the trained model to predict small ones. However, differences in gestational age as small as one week can significantly influence neonatal morbidity and mortality and long-term outcomes [18 – 23]. Hence, the model’s accuracy across the whole gestational age range becomes essential.
To solve this problem, we developed the R package eClock (ensemble-based clock). It improves the traditional machine learning strategy in handling the imbalance problem of category data [24], and combines bagging and SMOTE (Synthetic Minority Over-sampling Technique) methods to adjust the biased age distribution and predict DNAm age with an ensemble model. This is the first time applying these techniques to the clock model, bringing a new framework for clock model construction. eClock also provides other functions, such as training the traditional clock model, displaying features, and converting methylation probe/gene/DMR (DNA methylation region) values. To test the performance of the package, we used 3 different datasets, and the results show that the package can effectively improve the clock model performance on rare samples.
In this video we have a look at a case study of one person who has undergone hTERT gene therapy. The paper does not identify the subject I would guess it is Liz Parrish. The gene therapy was administered two times over a period of five years.
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Papers mentioned in the video:
Systemic Human Htert Aav Gene Transfer Therapy And The Effect On Telomere Length And Biological Age, A Case Report.
https://maplespub.com/article/systemic-human-htert-aav-gene-…ase-report.
Continue reading “35 Years Biological Age Reversal: A Case Study | Review” »
Scientists at the Max Planck Institute for the Science of Light (MPL) and Max-Planck-Zentrum für Physik und Medizin (MPZPM) in Erlangen present a large step forward in the characterization of nanoparticles. They used a special microscopy method based on interfereometry to outperform existing instruments. One possible application of this technique may be to identify illnesses.
Nanoparticles are everywhere. They are in our body as protein aggregates, lipid vesicles, or viruses. They are in our drinking water in the form of impurities. They are in the air we breath as pollutants. At the same time, many drugs are based on the delivery of nanoparticles, including the vaccines we have recently been given. Keeping with the pandemics, quick tests used for the detection the SARS-Cov-2 are based on nanoparticles too. The red line, which we monitor day by day, contains myriads of gold nanoparticles coated with antibodies against proteins that report infection.
Technically, one calls something a nanoparticle when its size (diameter) is smaller than one micrometer. Objects of the order of one micrometer can still be measured in a normal microscope, but particles that are much smaller, say smaller than 0.2 micrometers, become exceedingly difficult to measure or characterize. Interestingly, this is also the size range of viruses, which can become as small as 0.02 micrometers.
When cells reproduce, the internal mechanisms that copy DNA get it right nearly every time. Rice University bioscientists have uncovered a tiny detail that helps understand how the process could go wrong.
Their study of enzymes revealed the presence of a central metal ion critical to DNA replication also appears to be implicated in misincorporation, the faulty ordering of nucleotides on new strands.
A new two component-based glass/polydimethylsiloxane microfluidic pH-responsive carbon nanotube chip can efficiently capture or release cancer cells from blood samples.
CRISPR-Cas9 is considered a revolutionary gene editing tool, but its applications are limited by a lack of methods by which it can be safely and efficiently delivered into cells. Recently, a research team from Kumamoto University, Japan, have constructed a highly flexible CRISPR-Cas9 carrier using aminated polyrotaxane (PRX) that can not only bind with the unusual structure of Cas9 and carry it into cells, but can also protect it from intracellular degradation by endosomes.
Clustered regularly interspaced short palindromic repeats (CRISPR) and their accompanying protein, CRISPR-associated protein 9 (Cas9), made international headlines a few years ago as a game-changing genome editing system. Consisting of Cas9 and strand of genetic material known as a single-guide RNA (sgRNA), the system can target specific regions of DNA and function as “molecular scissors” to make precise edits. The direct delivery of Cas9–sgRNA complexes, i.e. Cas9 ribonucleoproteins (RNPs), into the nucleus of the cell is considered the safest and most efficient way to achieve genome editing. However, the Cas9 RNP has poor cellular permeability, and thus requires a carrier molecule to transport it past the first hurdle of the cell membrane before it can get to the cell nucleus. These carriers need to bind with Cas9 RNP, carry it into the cell, prevent its degradation by intracellular organelles called “endosomes,” and finally release it without causing any changes to its structure.
In a recent paper published in the June 2022, Volume 27 of Applied Materials Today, a research team from Kumamoto University has developed a transformable polyrotaxane (PRX) carrier that can facilitate genome editing using Cas9RNP with high efficiency and usability. “While there have been some PRX-based drug carriers for nucleic acids and proteins reported before, this is the first report on PRX-based Cas9 RNP carrier. Moreover, our findings describe how to precisely control intracellular dynamics across multiple steps. This will prove invaluable for future research in this direction,” says Professor Keiichi Motoyama, a corresponding author of the paper.
