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Gene Therapy: Is Still In Its Infancy But The Future Looks Promising

Gene therapy is the introduction of DNA into a patient to treat a genetic disease or a disorder. The newly inserted DNA contains a correcting gene to correct the effects of a disease, causing mutations. Gene therapy is a promising treatment for genetic diseases and also includes cystic fibrosis and muscular dystrophy. Gene therapy is a suitable treatment for infectious diseases, inherited disease and cancer.

Over the last few centuries, infectious diseases have been understood and tackled, through advances in sanitation, anti-microbial medications and vaccination. One day we may also be able to tackle genetic diseases – lifelong conditions arising from mutations that we inherit from our ancestors or that occur during our development.

Human gene editing is too transformative to be guided by the few

My editorial from today’s (3/18/19) Financial Times:

Far sooner than most people realise, the genetics revolution will transform the world within and around us. Although we think about genetic technologies primarily in the context of healthcare, these tools are set to change the way we make babies, the nature of the babies we make and, ultimately, our evolutionary trajectory as a species — and we are not remotely ready for what’s coming. Yet we must be, to optimise the benefits and minimise the potential harms of genetic technologies.

Scientists are now able to manipulate biology to a previously unimaginable degree. In the past year, we’ve seen two female mice having their own babies, dramatic increases in the precision of gene-editing tools, and the birth in China of the first gene-edited humans. As this science advances exponentially, however, the regulations guiding how it should best be used are struggling to keep up. If the applications race forward without appropriate guard rails, the danger increases that more scientists like He Jiankui, the Chinese biophysicist who genetically altered two girls, will put people’s health at risk. But if the regulations are hastily written before the issues are clear, are too strong or are not flexible enough, many people who would otherwise have benefited from applied genetic technologies will be condemned to unnecessary suffering or even death.

In World First, CRISPR Used on Patient’s Eye in Attempt to Cure Genetic Blindness

For the first time, doctors have attempted to cure blindness by gene-hacking a patient with CRISPR technology.

A team from Oregon Health & Science Institute injected three droplets of fluid that delivered the CRISPR DNA fragments directly into a patient’s eyeball, The Associated Press reports, in hopes that it will reverse a rare genetic condition called Leber congenital amaurosis, which causes blindness early in childhood.

“We literally have the potential to take people who are essentially blind and make them see,” Charles Albright, chief scientific officer of Editas Medicine, told the AP.

Gene-editing tool CRISPR used inside a human’s body for the first time, scientists say

Scientists say they have used the gene editing tool CRISPR inside someone’s body for the first time — offering a new frontier for efforts to operate on DNA, the chemical code of life, to treat diseases.

A patient recently had it done at the Casey Eye Institute at Oregon Health & Science University in Portland for an inherited form of blindness, according to the companies that make the treatment. The company would not give details on the patient or when the surgery occurred.

It may take up to a month to see if it worked to restore the patient’s vision. If the first few attempts seem safe, doctors plan to test it on 18 children and adults.

Our Genetic Future Is Coming… Faster Than We Think

If there was a public vote about human gene enhancement, would you vote YES or NO?


Our species is on the cusp of a revolution that will change every aspect of our lives but we’re hardly talking about it.

After three and a half billion years of evolution, two hundred and fifty thousand years of them as the ass-kicking bipedal hominins we call homo sapiens, we are on the verge of taking control of our evolutionary process unlike never before. This revolution will take hundreds of years to play out but it has already begun.

Sure, we influenced natural selection when we invented farming and modern medicine, but take a human baby from eleven thousand years ago and place him in a modern family and he’ll grow up just like any other kid. Then take a kid from a thousand years from now and place him in the same family. My belief is that the future child brought back to the present will not fit in nearly as well. He will be stronger and smarter with enhanced sensory and other capabilities. And we will have engineered him. We will have engineered us all.

CRISPR Scientists Hack Patient’s Genes in Bid to Cure Blindness

For the first time, doctors have attempted to cure blindness by gene-hacking a patient with CRISPR technology.

A team from Oregon Health & Science Institute injected three droplets of fluid that delivered the CRISPR DNA fragments directly into a patient’s eyeball, The Associated Press reports, in hopes that it will reverse a rare genetic condition called Leber congenital amaurosis, which causes blindness early in childhood.

“We literally have the potential to take people who are essentially blind and make them see,” Charles Albright, chief scientific officer of Editas Medicine, told the AP. Editas is one of the biotech companies that actually developed the treatment. “We think it could open up a whole new set of medicines to go in and change your DNA.”

Genome Assembly — The Holy Grail of Genome Analysis

The 2019 novel coronavirus or coronavirus disease (COVID-19) outbreak has threatened the entire world at present. Scientists are working day and night to understand the origin of COVID-19. You may have heard the news recently that the complete genome of COVID-19 has been published. How did scientists figure out the complete genome of COVID-19? In this article, I will explain how we can do this.

A genome is considered as all the genetic material, including all the genes of an organism. The genome contains all the information of an organism that is required to build and maintain it.

How can we read the information present in the genome? This is where sequencing comes into action. Assuming you have read my previous article on DNA analysis, you know that sequencing is used to determine the sequence of individual genes, full chromosomes or entire genomes of an organism.

Global Organization and Proposed Megataxonomy of the Virus World

Viruses and mobile genetic elements are molecular parasites or symbionts that coevolve with nearly all forms of cellular life. The route of virus replication and protein expression is determined by the viral genome type. Comparison of these routes led to the classification of viruses into seven “Baltimore classes” (BCs) that define the major features of virus reproduction. However, recent phylogenomic studies identified multiple evolutionary connections among viruses within each of the BCs as well as between different classes. Due to the modular organization of virus genomes, these relationships defy simple representation as lines of descent but rather form complex networks. Phylogenetic analyses of virus hallmark genes combined with analyses of gene-sharing networks show that replication modules of five BCs (three classes of RNA viruses and two classes of reverse-transcribing viruses) evolved from a common ancestor that encoded an RNA-directed RNA polymerase or a reverse transcriptase. Bona fide viruses evolved from this ancestor on multiple, independent occasions via the recruitment of distinct cellular proteins as capsid subunits and other structural components of virions. The single-stranded DNA (ssDNA) viruses are a polyphyletic class, with different groups evolving by recombination between rolling-circle-replicating plasmids, which contributed the replication protein, and positive-sense RNA viruses, which contributed the capsid protein. The double-stranded DNA (dsDNA) viruses are distributed among several large monophyletic groups and arose via the combination of distinct structural modules with equally diverse replication modules. Phylogenomic analyses reveal the finer structure of evolutionary connections among RNA viruses and reverse-transcribing viruses, ssDNA viruses, and large subsets of dsDNA viruses. Taken together, these analyses allow us to outline the global organization of the virus world. Here, we describe the key aspects of this organization and propose a comprehensive hierarchical taxonomy of viruses.

New insights into evolution: Why genes appear to move around

Scientists at Uppsala University have proposed an addition to the theory of evolution that can explain how and why genes move on chromosomes. The hypothesis, called the SNAP Hypothesis, is presented in the scientific journal PLOS Genet ics.

Life originated on Earth almost 4 billion years ago and diversified into a vast array of species. How did this diversification occur? The Theory of Evolution, together with the discovery of DNA and how it replicates, provide an answer and a mechanism. Mutations in DNA occur from generation to generation, and can be selected if they help individuals to adapt better to their environment. Over time, this has led to the separation of organisms into the different species that now inhabit all ecosystems.

Current theory holds that evolution involves mistakes made when replicating a gene. This explains how genes can mutate over time and acquire new functions. However, a mystery in biology is that the relative locations of genes on also change over time. This is obvious in bacteria, as different species often have the same genes in very different relative locations. Since the , genes have apparently been changing location. The questions are, how and why do genes move their relative locations?

Researchers catalog dozens of mutations in crucial brain development gene

An international team of researchers that pooled genetic samples from developmentally disabled patients from around the world has identified dozens of new mutations in a single gene that appears to be critical for brain development.

“This is important because there are a handful of that are recognized as ‘hot spots’ for causing ,” said lead author Debra Silver, an associate professor of molecular genetics and microbiology in the Duke School of Medicine. “This gene, DDX3X, is going to be added to that list now.”

An analysis led by the Elliott Sherr lab at the University of California-San Francisco found that half of the DDX3X mutations in the 107 children studied caused a loss of function that made the gene stop working altogether, but the other half caused changes predicted to disrupt the function of the gene.