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A few days ago, the world-renowned technology media "MIT Technology Review" (MIT Technology).
Review) announced its "Top 10 Global Breakthrough Technologies" for 2023, with three unique seats in the life sciences sector
.
They will have a significant impact on our lives, promising not only to bring once-and-for-all treatments for common diseases, but also to solve the global problem
of transplant organ shortages.
of transplanted organ shortages.
Breakthrough of the Year: Gene editing lowers high cholesterol
Breakthrough of the Year: Gene editing lowers high cholesterolRelated institutions: Verve Therapeutics, Beam Therapeutics, Prime Medicine, Broad Institute
Related institutions: Verve Therapeutics, Beam Therapeutics, Prime Medicine, Broad InstituteLast year, a woman in New Zealand became the first patient
to have her cholesterol levels permanently lowered through gene-editing therapy.
The MIT Technology Review article points out that this could be a turning point
for CRISPR gene-editing technology.
Since the advent of the CRISPR gene-editing tool, its application has shifted from the laboratory to clinical development, but the first therapies in the pipeline have focused on rare genetic diseases
.
Therapies to lower high cholesterol have broader potential
.
in CRISPR gene-editing technology.
This cholesterol-lowering gene editing therapy was developed by Verve
Developed by Therapeutics, it is based on single-base editing technology, which is more precise
than CRISPR.
Single-base editing can complete precise gene editing
without cutting the DNA double strand.
If CRISPR-Cas9 technology is "scissors" that modify the genome, single-base editing methods are "pencils and erasers" that erase and rewrite a letter
in a gene.
In theory, it may reduce off-target effects and lead to greater safety
.
without cutting the DNA double strand.
If CRISPR-Cas9 technology is "scissors" that modify the genome, single-base editing methods are "pencils and erasers" that erase and rewrite a letter
in a gene.
Image source: Verve's official website
Prime Medicine, co-founded by Dr.
Liu Ruqian, is a step further and is called Prime Editor
editing) not only converts any base to another, but also inserts DNA fragments into the genome
precisely.
This makes it possible to
replace the genes that cause the disease.
precisely.
This makes it possible to replace the genes that cause the disease
The pilot editing system can not only convert between any bases, but also insert or delete specific DNA sequences (Image source: Prime Medicine's website)
The pilot editing system can not only convert between any bases, but also insert or delete specific DNA sequences (Image source: Prime Medicine's website)These new gene-editing technologies promise to expand the range of diseases that gene editing can treat, and they are no longer limited to genetic diseases, and one day it may be possible to prevent high blood pressure or other diseases by adding gene fragments to the genome, which may benefit a wide range of people
around the world.
around the world.
However, industry experts also pointed out that expanding the scope of application of gene editing to treat diseases requires a comprehensive measurement
of the benefit/risk ratio of gene editing therapy.
At the just-concluded 2023 WuXi AppTec Global Forum, Joseph, director of the Stanford Cardiovascular Institute and new president of the American Cardiovascular Association
Asked about the prospect of using gene editing to lower cholesterol, Dr.
Wu said it makes sense to use gene editing to lower cholesterol in patients at high risk of cardiovascular disease all at once
.
The use of gene editing in a broader patient population requires comparing the benefit/risk ratio of gene editing with currently available lipid-lowering therapies such as statins
.
Gene editing has the potential
for lifelong benefits in one treatment.
At the tipping point for gene therapy to benefit the public, the long-term effects of its potential off-target effects also need to be evaluated
with sufficient clinical data.
of the benefit/risk ratio of gene editing therapy.
Gene editing has the potential
for lifelong benefits in one treatment.
At the tipping point for gene therapy to benefit the public, the long-term effects of its potential off-target effects also need to be evaluated
with sufficient clinical data.
Joseph Joseph, director of the Stanford Cardiovascular Institute and the new president of the American Cardiovascular Association
Dr.
Wu shared insights on new drug development for cardiovascular disease at the WuXi AppTec Global Forum 2023
Wu shared insights on new drug development for cardiovascular disease at the WuXi AppTec Global Forum 2023
Breakthrough of the Year: Making the Transplant Shortage a Thing of the Past
Breakthrough of the Year: Making the Transplant Shortage a Thing of the PastRelated institutions: eGenesis, Makana Therapeutics, United Therapeutics
Related institutions: eGenesis, Makana Therapeutics, United TherapeuticsDavid, 57, 2022
Mr.
Bennett became the first patient
to receive a pig heart transplant.
With this work, researchers at the University of Maryland have taken a crucial step
in exploring whether genetically edited pig hearts can sustain human life.
Globally, about 130,000 patients receive organ transplants each year to save their lives, yet far more die because they can't wait for the right organ
.
One strategy to address this challenge is the use of animal organs, however a major challenge that xenotransplantation needs to overcome is the body's immune rejection
.
At present, several biotechnology companies are using gene editing to modify pigs, eliminate the antigens that stimulate the human immune response, and add other genes to make pig organs more similar
to human organs.
The pig heart implanted into Mr.
Bennett contained 10 gene edits
.
Although Mr.
Bennett died two months after receiving a pig heart transplant, the researchers did not observe typical immune rejection
.
And the discovery of porcine cytomegalovirus in pig hearts may have adverse effects
on transplant outcomes.
Based on these lessons, the researchers plan to conduct clinical trials in more patients to explore the potential
of xenotransplantation.
Bennett contained 10 gene edits
.
Although Mr.
Bennett died two months after receiving a pig heart transplant, the researchers did not observe typical immune rejection
.
At the just-concluded WuXi AppTec Global Forum, Juergen, senior vice president and head of Bayer's Leaps
Looking to the future, Dr.
Eckhardt also believes that in ten years' time, xenotransplantation will become a reality, and patients who need to transplant organs will no longer have to wait
.
Eckhardt also believes that in ten years' time, xenotransplantation will become a reality, and patients who need to transplant organs will no longer have to wait
.
Juergen, senior vice president and head of Bayer Leaps
Dr.
Eckhardt looked forward to the development of medicine in 10 years at the 2023 WuXi AppTec Global Forum
Eckhardt looked forward to the development of medicine in 10 years at the 2023 WuXi AppTec Global Forum
In addition, researchers are using human cells to build complex tissues and organs from scratch, some 3D printing technologies have been able to build the basic structure of the lung, and other engineering techniques can grow small organoids based on stem cells that can mimic the function of
specific human organs.
specific human organs.
The MIT Technology Review article points out that whether it is originated in animals or built from human cells in factories, a steady supply of organs can make transplants more common and allow more patients to be given a new life
.
Breakthrough of the Year: Analyzing Ancient DNA
Breakthrough of the Year: Analyzing Ancient DNAAssociated institutions: Max Planck Institute for Evolutionary Anthropology, Laboratory of Dr.
David Reich, Harvard University
David Reich, Harvard University
In the past, scientists had to look for teeth or bones from many ancient human remains to find samples that were intact enough to analyze
.
However, with the development of more innovative and economical sequencing technology, decrypting damaged DNA is no longer difficult, and a new page
has opened in the field of ancient DNA analysis.
Today, scientists can even analyze tiny amounts of DNA in soil stained with Neanderthal urine, eliminating the need to extract DNA
from teeth or bones.
The new field, known as paleogenetics, was also followed by Svante last November
Dr.
Pääbo is widely known
for winning the Nobel Prize in Physiology or Medicine.
Pääbo is widely known for winning the Nobel Prize in Physiology or Medicine
Reference reading: The Nobel Prize in Physiology or Medicine has just been announced! Straight to the ultimate human question: Why are we unique?
Advances in ancient DNA analysis techniques have allowed scientists to obtain vast amounts of data
on ancient human genes.
In 2010, scientists had only 5 samples of ancient human whole genome data
.
However, this value has grown by leaps and bounds in recent years, and scientists have obtained genome-wide data
on 5550 ancient humans in 2020.
The breakthrough also led scientists to discover two extinct human races, the Luzon (Homo).
luzonensis) and Denisovans, and found that modern humans actually carry a large number of Denisovan and Neanderthal genes
.
luzonensis) and Denisovans, and found that modern humans actually carry a large number of Denisovan and Neanderthal genes
Dr.
Päääbo extracted DNA
from bone samples from extinct humans.
He first obtained a fragment of bone from Neanderthal, Germany, and later analyzed it using bone fragments from the Denisovan cave
.
The evolutionary tree shows the relationship between Homo sapiens and these extinct ancient humans, as well as the transfer between genes discovered by Dr Päpbo (Image source: Reference [2])
Päääbo extracted DNA
from bone samples from extinct humans.
He first obtained a fragment of bone from Neanderthal, Germany, and later analyzed it using bone fragments from the Denisovan cave
.
The evolutionary tree shows the relationship between Homo sapiens and these extinct ancient humans, as well as the transfer between genes discovered by Dr Päpbo (Image source: Reference [2])
In addition to advances in archaeology, ancient DNA analysis techniques have also solved mysteries that historians had no way of knowing in the past
.
For example, the analysis confirms that the ancestors of the Indian population actually come from many different and mixed backgrounds, a finding that reduces the impact
of the caste system on society.
Moreover, analysis of DNA from the battlefield of Sicily 2,500 years ago found that the composition of the ancient Greek army was far more complex and diverse
than historians have depicted in the past.
Not only that, but the analysis of these ancient samples also answers the mysteries related to modern health
.
Last year, scientists identified a mutation that improved survival against the Black Death by 40 percent — but also increased the number of individuals with the mutation with diseases like Crohn's
disease) and the risk
of autoimmune diseases.
Perhaps the attitudes of different cultures toward human remains will influence what scholars want to explore in ancient DNA research, but there is no doubt that the revelations of ancient DNA have rewritten our understanding
of history.
Resources:
Resources:
[1] 10 breakthrough technologies 2023.
Retrieved January 11, 2023, from
Retrieved January 11, 2023, from [2] The Nobel Prize in Physiology or Medicine 2022, Retrieved October 3, 2022, from [2] The Nobel Prize in Physiology or Medicine 2022, Retrieved October 3, 2022, from