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Recent popular reports from Yimike ★Xu Congcong, head of research on the microbial mRNA platform: Nucleic acid drugs are the third milestone in drug development, and we look forward to the combined treatment strategies of different technical routes.
★Lysogene completed the first case of AAV gene therapy for GM1 gangliosidosis Patient medicationMedClub broke the news, click on the picture, and register now June 15, 2021/MedClub News/--In 2020, CRISPR/Cas9 technology successfully won the Nobel Prize, becoming the most popular gene editing technology at the moment
.
CRISPR/Cas9 has strong potential in the medical field and has shown strong therapeutic effects in a variety of genetic diseases
.
In March 2021, Integrated DNA Technologies (IDT), together with researchers from world-renowned institutions such as Stanford University and Baylor College of Medicine, published a major study in the top international academic journal Nature Medicine (impact factor: 36.
130) The thesis, using IDT's Alt-R® CRISPR/Cas9 gene editing system and Lonza's electrotransfection system, successfully used β-globin instead of α-globin to treat β-thalassemia in a mouse model
.
▲ Image source: Nature Medicine In this study, researchers used LONZA 4D-Nucleofector to electroporate CD34+ HSPC from β-thalassaemia, and then used AAV6 as a carrier to deliver IDT’s Cas9 combined with chemically synthesized sgRNA to HSPC for processing Gene editing, while maintaining the highly homologous HBA2 gene, knock out the endogenous HBA1 gene and replace it with the full-length HBB gene
.
▲ Picture source: Nature Medicine research results show that this gene editing strategy normalizes the ratio of β-globin:α-globin messenger RNA to protein, and can produce functional adult hemoglobin tetramers; inject back in a mouse model Later, the gene-edited HSPC can carry out long-term and two-line hematopoietic reconstitution in mice
.
It is suggested that HBB can replace HBA1 as a new treatment strategy for β-thalassemia
.
The researchers said that this study may be the first to prove that the entire endogenous gene replacement can be achieved under Cas9-mediated DNA double-strand break (DSB)
.
This discovery is expected to expand the application scope of CRISPR/Cas9 to treat more types of diseases caused by gene mutations
.
Breaking through the limitations of traditional CRISPR/Cas9 In recent years, CRISPR gene editing technology has been widely used in many fields.
It is foreseeable that the transformation of CRISPR-based genome editing technology into medical applications will have a significant impact on human health, and this technology has also led A revolution in molecular biology tools
.
However, CRISPR/Cas9 still has some limitations, which limit the rapid development of CRISPR/Cas9, such as off-target effects
.
At the same time, the traditional CRISPR/Cas technology has powerful advantages in gene cutting, but there are still certain challenges in the precise targeted insertion/replacement of long DNA fragments
.
In order to overcome these problems, IDT has developed the Alt-R® CRISPR/Cas9 gene editing system, which reduces the overall off-target rate while achieving efficient cutting at the target site
.
At the same time, the newly published research shows that this new CRISPR/Cas9 system can insert/replace pathogenic genes with long fragments of functional genes
.
The Alt-R® CRISPR/Cas9 editing system mainly uses the method of delivering ribonucleoprotein complex (RNP).
RNP is a complex with editing function formed by the combination of Cas9 protein and gRNA
.
Unlike traditional CRISPR/Cas9, in IDT's Alt-R® CRISPR system, the components of gRNA, crRNA and tracrRNA, are optimized
.
In fact, another research article published in the journal Nature Medicine in 2018 confirmed the superior performance of Alt-R® CRISPR/Cas9
.
In this study, IDT's Alt-R® SP HiFi Cas9 showed stable target editing activity with extremely low miss rate
.
▲ Picture source: Nature Medicine research shows: HiFi Cas9/AAV6 can induce powerful gene editing at 5 treatment sites (HBB, IL2RG, CCR5, HEXB, TRAC), with high targeting; at the same time, HiFi Cas9/AAV6 mediates High level correction of p.
E6V mutation in HSPC of patients with sickle cell disease (SCD)
.
IDT's Alt-R products make CRISPR experiments no longer difficult.
In the above studies, IDT's Alt-R products are used to help customers better complete their research
.
In the field of gene editing, IDT can provide corresponding Alt-R CRISPR design solutions according to the different needs of customers
.
Design different guide RNAs according to customer needs: Cas9 crRNA: tracrRNA, Cas9 sgRNA, Cas12a crRNA; a series of Alt-R CRISPR Cas enzymes including WT Cas9, HiFi Cas9, Cas9 nickases, Cas12a (Cpf1), Cas12a (Cpf1) Ultra ; And Alt-R electroporation enhancer
.
IDT can also provide a series of Alt-R HR reagents
.
Through chemically synthesized highly customized guide RNA (gRNA) sequences, supporting electroporation/knock-in enhancers, various control reagents, and free-to-use sequence design tools, the entire process from experimental design to result analysis is covered.
Realize efficient and accurate gene editing, making experiments no longer difficult
.
Integrated DNA Technologies, Inc.
(IDT) is an operating company under the life sciences platform of Danaher Corporation (NYSE: DHR)
.
IDT is committed to the development, production and sales of nucleic acid products for the life science community, and provides support for academic and commercial research, agriculture, medical diagnosis, and drug development
.
IDT has developed a number of patented technologies for genome applications, covering next-generation sequencing, CRISPR genome editing, synthetic biology, digital PCR, and RNA interference
.
Through GMP services, IDT products are used by scientists to study various forms of cancer and various genetic and infectious diseases, provide services for more than 130,000 life science researchers, and are recognized as a leader in the custom nucleic acid production industry
.
Lonza's Nucleofector technology easily achieves efficient CRISPR transduction.
CRISPR technology is another effective tool for site-specific genome modification in addition to ZFN and TALEN technologies
.
How to efficiently transduce the CRISPR system into target cells has become another problem that needs to be solved urgently
.
Lonza's unique Nucleofector technology has a history of 20 years and has been widely recognized by customers since its inception in 2001.
It has been proven to be an effective non-viral transfection method based on CRISPR-based genome editing tools
.
Nucleofector technology uses a combination of pre-optimized electroporation parameters and cell type-specific electroporation solutions to enable the substrate to be directly transfected into the nucleus
.
Compared with traditional electroporation, direct transfection into the nucleus has nothing to do with cell division, and has a higher transfection efficiency even in non-dividing primary cells
.
Nucleofector technology is modularized for small-volume transfection and large-volume transfection, which can realize linear amplification from small volume to large volume, and is suitable for the transformation from the drug development stage to the clinical stage
.
Headquartered in Basel, Switzerland, Lonza is an excellent partner in the global pharmaceutical, biotechnology and nutrition markets
.
Lonza is committed to disease prevention and promotes a healthier world by helping customers launch innovative drugs to assist in the treatment of a series of diseases
.
Lonza perfectly combines advanced manufacturing, scientific expertise, excellent craftsmanship and technical insight to achieve its goals and help customers commercialize their discoveries and innovations in the medical and health field
.
Lonza serves many customers, from large biotechnology and pharmaceutical companies, to academic institutions and government departments, providing them with advanced production services from preclinical, clinical to commercialization, and also providing them with innovative research tools, and Solutions that support the commercialization of products throughout the process, covering the research and development and production of cell gene therapy, antibody drugs, vaccines and other fields
.
This article is based on the analysis of relevant literature, and does not intend to make any recommendations or guarantees about the commerciality or applicability of the said products or services.
The introduction of the said products or services, including data and pictures, has been sorted and published by specific entrustment/authorization.
.
Reference materials: 1.
https:// /sg.
idtdna.
com/pages/products/crispr-genome-editing
★Lysogene completed the first case of AAV gene therapy for GM1 gangliosidosis Patient medicationMedClub broke the news, click on the picture, and register now June 15, 2021/MedClub News/--In 2020, CRISPR/Cas9 technology successfully won the Nobel Prize, becoming the most popular gene editing technology at the moment
.
CRISPR/Cas9 has strong potential in the medical field and has shown strong therapeutic effects in a variety of genetic diseases
.
In March 2021, Integrated DNA Technologies (IDT), together with researchers from world-renowned institutions such as Stanford University and Baylor College of Medicine, published a major study in the top international academic journal Nature Medicine (impact factor: 36.
130) The thesis, using IDT's Alt-R® CRISPR/Cas9 gene editing system and Lonza's electrotransfection system, successfully used β-globin instead of α-globin to treat β-thalassemia in a mouse model
.
▲ Image source: Nature Medicine In this study, researchers used LONZA 4D-Nucleofector to electroporate CD34+ HSPC from β-thalassaemia, and then used AAV6 as a carrier to deliver IDT’s Cas9 combined with chemically synthesized sgRNA to HSPC for processing Gene editing, while maintaining the highly homologous HBA2 gene, knock out the endogenous HBA1 gene and replace it with the full-length HBB gene
.
▲ Picture source: Nature Medicine research results show that this gene editing strategy normalizes the ratio of β-globin:α-globin messenger RNA to protein, and can produce functional adult hemoglobin tetramers; inject back in a mouse model Later, the gene-edited HSPC can carry out long-term and two-line hematopoietic reconstitution in mice
.
It is suggested that HBB can replace HBA1 as a new treatment strategy for β-thalassemia
.
The researchers said that this study may be the first to prove that the entire endogenous gene replacement can be achieved under Cas9-mediated DNA double-strand break (DSB)
.
This discovery is expected to expand the application scope of CRISPR/Cas9 to treat more types of diseases caused by gene mutations
.
Breaking through the limitations of traditional CRISPR/Cas9 In recent years, CRISPR gene editing technology has been widely used in many fields.
It is foreseeable that the transformation of CRISPR-based genome editing technology into medical applications will have a significant impact on human health, and this technology has also led A revolution in molecular biology tools
.
However, CRISPR/Cas9 still has some limitations, which limit the rapid development of CRISPR/Cas9, such as off-target effects
.
At the same time, the traditional CRISPR/Cas technology has powerful advantages in gene cutting, but there are still certain challenges in the precise targeted insertion/replacement of long DNA fragments
.
In order to overcome these problems, IDT has developed the Alt-R® CRISPR/Cas9 gene editing system, which reduces the overall off-target rate while achieving efficient cutting at the target site
.
At the same time, the newly published research shows that this new CRISPR/Cas9 system can insert/replace pathogenic genes with long fragments of functional genes
.
The Alt-R® CRISPR/Cas9 editing system mainly uses the method of delivering ribonucleoprotein complex (RNP).
RNP is a complex with editing function formed by the combination of Cas9 protein and gRNA
.
Unlike traditional CRISPR/Cas9, in IDT's Alt-R® CRISPR system, the components of gRNA, crRNA and tracrRNA, are optimized
.
In fact, another research article published in the journal Nature Medicine in 2018 confirmed the superior performance of Alt-R® CRISPR/Cas9
.
In this study, IDT's Alt-R® SP HiFi Cas9 showed stable target editing activity with extremely low miss rate
.
▲ Picture source: Nature Medicine research shows: HiFi Cas9/AAV6 can induce powerful gene editing at 5 treatment sites (HBB, IL2RG, CCR5, HEXB, TRAC), with high targeting; at the same time, HiFi Cas9/AAV6 mediates High level correction of p.
E6V mutation in HSPC of patients with sickle cell disease (SCD)
.
IDT's Alt-R products make CRISPR experiments no longer difficult.
In the above studies, IDT's Alt-R products are used to help customers better complete their research
.
In the field of gene editing, IDT can provide corresponding Alt-R CRISPR design solutions according to the different needs of customers
.
Design different guide RNAs according to customer needs: Cas9 crRNA: tracrRNA, Cas9 sgRNA, Cas12a crRNA; a series of Alt-R CRISPR Cas enzymes including WT Cas9, HiFi Cas9, Cas9 nickases, Cas12a (Cpf1), Cas12a (Cpf1) Ultra ; And Alt-R electroporation enhancer
.
IDT can also provide a series of Alt-R HR reagents
.
Through chemically synthesized highly customized guide RNA (gRNA) sequences, supporting electroporation/knock-in enhancers, various control reagents, and free-to-use sequence design tools, the entire process from experimental design to result analysis is covered.
Realize efficient and accurate gene editing, making experiments no longer difficult
.
Integrated DNA Technologies, Inc.
(IDT) is an operating company under the life sciences platform of Danaher Corporation (NYSE: DHR)
.
IDT is committed to the development, production and sales of nucleic acid products for the life science community, and provides support for academic and commercial research, agriculture, medical diagnosis, and drug development
.
IDT has developed a number of patented technologies for genome applications, covering next-generation sequencing, CRISPR genome editing, synthetic biology, digital PCR, and RNA interference
.
Through GMP services, IDT products are used by scientists to study various forms of cancer and various genetic and infectious diseases, provide services for more than 130,000 life science researchers, and are recognized as a leader in the custom nucleic acid production industry
.
Lonza's Nucleofector technology easily achieves efficient CRISPR transduction.
CRISPR technology is another effective tool for site-specific genome modification in addition to ZFN and TALEN technologies
.
How to efficiently transduce the CRISPR system into target cells has become another problem that needs to be solved urgently
.
Lonza's unique Nucleofector technology has a history of 20 years and has been widely recognized by customers since its inception in 2001.
It has been proven to be an effective non-viral transfection method based on CRISPR-based genome editing tools
.
Nucleofector technology uses a combination of pre-optimized electroporation parameters and cell type-specific electroporation solutions to enable the substrate to be directly transfected into the nucleus
.
Compared with traditional electroporation, direct transfection into the nucleus has nothing to do with cell division, and has a higher transfection efficiency even in non-dividing primary cells
.
Nucleofector technology is modularized for small-volume transfection and large-volume transfection, which can realize linear amplification from small volume to large volume, and is suitable for the transformation from the drug development stage to the clinical stage
.
Headquartered in Basel, Switzerland, Lonza is an excellent partner in the global pharmaceutical, biotechnology and nutrition markets
.
Lonza is committed to disease prevention and promotes a healthier world by helping customers launch innovative drugs to assist in the treatment of a series of diseases
.
Lonza perfectly combines advanced manufacturing, scientific expertise, excellent craftsmanship and technical insight to achieve its goals and help customers commercialize their discoveries and innovations in the medical and health field
.
Lonza serves many customers, from large biotechnology and pharmaceutical companies, to academic institutions and government departments, providing them with advanced production services from preclinical, clinical to commercialization, and also providing them with innovative research tools, and Solutions that support the commercialization of products throughout the process, covering the research and development and production of cell gene therapy, antibody drugs, vaccines and other fields
.
This article is based on the analysis of relevant literature, and does not intend to make any recommendations or guarantees about the commerciality or applicability of the said products or services.
The introduction of the said products or services, including data and pictures, has been sorted and published by specific entrustment/authorization.
.
Reference materials: 1.
https:// /sg.
idtdna.
com/pages/products/crispr-genome-editing
