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Author | Wang Cong
Author | Wang CongEdit | Wang Duoyu
Typography | Hydrography
Typography | Hydrography
The blood-brain barrier (BBB) refers to the barrier between blood plasma and brain cells formed by brain capillary walls and glial cells, and the barrier between plasma and cerebrospinal fluid formed by the choroid plexus.
Only specific types of molecules are allowed to pass from the blood.
The flow enters the brain neurons and other surrounding cells
.
Blood - brain barrier The blood - brain barrier (BBB) refers to the barrier between blood plasma and brain cells formed by brain capillary walls and glial cells, and the barrier between plasma and cerebrospinal fluid formed by the choroid plexus.
The existence of the blood-brain barrier is of great significance in preventing harmful substances from entering the brain from the blood.
The existence of the blood-brain barrier is of great significance in preventing harmful substances from entering the brain from the blood.
With the increasingly serious population aging problem, neurodegenerative diseases such as Alzheimer’s disease, Huntington’s disease, Parkinson’s disease are growing rapidly, and the treatment of brain diseases is facing severe challenges.
With the increasingly serious population aging problem, neurodegenerative diseases such as Alzheimer’s disease, Huntington’s disease, Parkinson’s disease are growing rapidly, and the treatment of brain diseases is facing severe challenges.
Recently, the California Institute of TechnologyViviana Gradinaruteam inNature Neurosciencejournal published a report entitled: AAV capsid variants with Transgene expression The Brain-Wide and Decreased liver Targeting the After intravenous Delivery in Mouse and marmosetresearch papers
Recently, the California Institute of TechnologyViviana Gradinaru team in Nature Neuroscience journal published a report entitled: Viviana Gradinaru Nature Neuroscience AAV capsid variants with Transgene expression The Brain-Wide and Decreased liver Targeting the After intravenous Delivery in Mouse and marmoset research papers
The research developed a new adeno-associated virus (AAV) variant - AAV.
The research and development of a novel adeno-associated virus (AAV) variant - AAV.
Adeno-associated virus (AAV) has long been regarded as the most promising gene therapy delivery vector, and it has been clinically verified
Adeno-associated virus Adeno-associated virus (AAV) has long been regarded as the most promising gene therapy delivery vector, and it has been clinically verified
AAV is composed of two main parts: a capsid made of protein ; and genetic material wrapped in the capsid
AAV is composed of two main parts: a capsid made of protein ; and genetic material wrapped in the capsid
The Viviana Gradinaru team at the California Institute of Technology has been working on the development and design of AAV capsids that can cross the blood-brain barrier for nearly a decade, and enable it to specifically target specific cell types in the brain
The Viviana Gradinaru team at the California Institute of Technology has been working on the development and design of AAV capsids that can cross the blood-brain barrier for nearly a decade, and enable it to specifically target specific cell types in the brain
.
Previously, the team developed AAV-PHP.
eB, which can efficiently cross the blood-brain barrier through intravenous injection, but at the same time it will be enriched in the liver
.
Viviana Gradinaru
In the new study, conducted by the team of AAV9 capsid transformation, developed a series of AAV variants, which AAV.
CAP-B10 , not only can effectively cross the blood-brain barrier into the brain cells, particularly neurons, while It can also include systemic targets including the liver
.
This neuron-specific targeting and low liver targeting across the blood-brain barrier has been confirmed not only in mice, but also in non-human primates, marmosets
.
CAP-B10 , not only can effectively cross the blood-brain barrier into the brain cells, particularly neurons, while It can also include systemic targets including the liver
.
This neuron-specific targeting and low liver targeting across the blood-brain barrier has been confirmed not only in mice, but also in non-human primates, marmosets
.
AAV.
CAP-B10 can not only efficiently cross the blood-brain barrier and enter brain cells, especially neurons, but also systemic targets including the liver.
Prior to this, the AAV-PHP.
eB and other vectors developed by the team were unsuccessful in non-human primate experiments, and this new study looked for the AAV.
CAP-B10 developed in non-human primates.
The same effect is significant in the medium, compared with AAV9 and AAV-PHP.
eB, AAV.
CAP-B10 can specifically target neurons, and will not be enriched in the liver
.
eB and other vectors developed by the team were unsuccessful in non-human primate experiments, and this new study looked for the AAV.
CAP-B10 developed in non-human primates.
The same effect is significant in the medium, compared with AAV9 and AAV-PHP.
eB, AAV.
CAP-B10 can specifically target neurons, and will not be enriched in the liver
.
AAV.
CAP-B10
Viviana Gradinaru's team modified the AAV capsid at multiple sites through directed evolution, and produced a variety of AAV variants that can cross the blood-brain barrier in mice and monkeys
.
These findings also indicate that modifications to the AAV capsid can improve the delivery efficiency of gene therapy and increase the specificity of neuron targeting
.
This new AAV vector also provides a safer and more effective treatment option for brain diseases
.
.
These findings also indicate that modifications to the AAV capsid can improve the delivery efficiency of gene therapy and increase the specificity of neuron targeting
.
This new AAV vector also provides a safer and more effective treatment option for brain diseases
.
This new AAV vector also provides a safer and more effective treatment option for brain diseases
Viviana Gradinaru
Viviana Gradinaru
Viviana Gradinaru is Zhang Feng ’s younger sister.
Both graduated with a Ph.
D.
from the laboratory of Professor Karl Deisseroth , the father of optogenetics .
During the Ph.
D.
period, Viviana and Zhang Feng published several important papers in the field of optogenetics
.
Both graduated with a Ph.
D.
from the laboratory of Professor Karl Deisseroth , the father of optogenetics .
During the Ph.
D.
period, Viviana and Zhang Feng published several important papers in the field of optogenetics
.
Viviana Gradinaru Zhang Feng Karl Deisseroth Viviana Zhang Feng
Paper link:
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