Harvard University: A revolutionary DNA base editing technology for development and commercialization.

Harvard University announced Thursday that it will grant beam therapeutics (BT) a global patent license to develop and commercialize a revolutionary set of DNA base editing technologies that can be used to treat human diseases.
BT announced on the same day that it had raised up to $87 million in a round of a funding led by F-Prime Capital and ARCH Ventures.
BT was co-founded by Gene Editing Technology Leaders Liu Ruqian, Zhang Feng and Keith Yang. "Base editing represents a powerful platform for solving a broad class of genetic diseases that can be solved with other genome editing methods," said Vivian Bolling, director of business development at Harvard University's Technology Development Office at the
.
" reporters learned that licensed technology platforms include base editing and technologies related to the enhancement of base editing target range, opening the door to the use of gene editing technology to treat a range of human genetic diseases.
CRISPR Gene Editing: It is a godander but there are short plates in the human genome, and tens of thousands of mutations in DNA base (A, C, G, T) sequences are known to cause disease.
most disease-related human DNA variants consist of site mutations, or a single base pair in the genome is replaced by another base pair.
this mutation has been demonstrated in studies of various genetic diseases such as neurodegenerative diseases, metabolic diseases, blood diseases, vision or hearing loss.
genome editing techniques using the CRISPR platform and combined with Cas9 and Cpf1 enzymes have shown great promise of regulating genes by inserting or removing multiple nucleotides, but this technique is difficult to correct a single nucleotide cleanly and effectively.
existing genome editing methods use CRISPR as molecular scissors for double-stranded fracture, and then rely on the introduction of DNA templates for guided correction, trying to correct point mutations.
however, a double-stranded break within a cell triggers a process of reconnecting the end of the fracture and leads to the negative effects of random insertion and deletion.
therefore, the precise correction of point mutations usually has to compete with these undesirable by-products.
In addition, accurate correction using CRISPR/Cas9 usually relies on cell components, which are missing in inactive lyses cells, most of the body's cells.
base editing technology: Point-to-point precision target base editing technology multi-functional platform by Harvard University Professor of Chemistry and Chemical Biology Liu Ruqian led the invention.
base editing technique is not to precisely correct disease-causing mutations in a particular gene, but to cut more targets that destroy genes or create mutations.
programmable molecular machine developed by Liu Ruqian's team, it can enter the target site in the selected cell genome DNA, directly converting one base into another, without producing double-stranded fractures in the DNA.
the technology uses an engineered multi-component protein containing improved Cas9 to unlock the targeted part of the DNA spiral and open a small window for operation on a single base without causing the double-stranded fracture in the DNA.
then, base editing directly converts the target gene from a mutated form to a correction form and, in some cases, requires the addition of a protein component to prevent the cell from uncorrecting the correction.
at the same time, the modified Cas9 cut unedited DNA strands, prompting the cell to repair a second chain with a base that complements the modified base. the result of
is that the double exchange will permanently place the entire base pair (e.g. A. T) to a different base pair (e.g. G. C).
Over the past year and a half, Liu's team has greatly expanded the scope of base editing technology.
expandthe targeting range, improve target DNA specificity, and create new base editors, which will have a significant impact on the treatment of genetic diseases.
ultimate goal: to fundamentally change medicine, Liu Ruqian said: "The ultimate goal is to be able to change the DNA base into another DNA base at will, cleanly and efficiently in an unmodified organism, whether human, plant or animal." "Our goal is to develop this innovative technology into the most extensive and transformative treatment for human diseases," said Mr Bolling,
.
licensing start-ups for commercial development ensures rapid resource mobilization and the full development and utilization of new technologies in this area.
" BT CEO John Evans points out that base editing technology can be modified in a single base with efficient and unprecedented control, and that BT will combine key technologies for base editing to create a wide range of precision gene drug pipelines, repair disease-causing site mutations, write protective genetic mutations, or regulate the expression or function of disease-causing genes, and ultimately use base editing as a treatment option for human diseases. "Genomic editing and base editing technologies fully reflect the continued and important contribution of Harvard university researchers to biomedical innovation and the opportunities that this progress creates for economic development and social benefits," said Isaac Colberg, senior assistant dean and chief technology development officer at Harvard University in
.
this opportunity is not only aimed at addressing disease treatment, but may also fundamentally change the practice of medicine.
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