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PubMed Journals Articles About "Genome Editing Startup Beam Therapeutics Already Trying Public" RSS

17:59 EST 15th December 2019 | BioPortfolio

Genome Editing Startup Beam Therapeutics Already Trying Public PubMed articles on BioPortfolio. Our PubMed references draw on over 21 million records from the medical literature. Here you can see the latest Genome Editing Startup Beam Therapeutics Already Trying Public articles that have been published worldwide.

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Showing "Genome editing startup Beam Therapeutics already trying public" PubMed Articles 1–25 of 10,000+

Perspectives on the application of genome editing technologies in crop breeding.

Most conventional and modern crop improvement methods exploit natural or artificially induced genetic variations and require laborious characterization of multiple generations of time-consuming genetic crosses. Genome editing systems, in contrast, provide the means to rapidly modify genomes in a precise and predictable way, making it possible to introduce improvements directly into elite varieties. Here, we describe the range of applications available to agricultural researchers using existing genome editin...


CRISPR/CAS9 Gene-Editing Strategies in Cardiovascular Cells.

Cardiovascular diseases are among the main causes of morbidity and mortality in Western countries and considered as a leading public health issue. Therefore, there is a strong need for new disease models to support the development of novel therapeutics approaches. The successive improvement of genome editing tools with zinc finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs), and more recently with clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associ...

Use of Genome Editing Tools in Environmental Health Research.

The nature and types of genome editing tools are rapidly expanding and becoming increasingly incorporated into research efforts aimed at understanding human disease. The majority of research involving genome editing has been driven by medical research, with a limited but increasing number of studies currently published in the field of environmental health and toxicology. This review aims to address this research gap by providing a high-level summary of current genome editing techniques and presenting exampl...


Genome Editing and Hematologic Malignancy.

The modern genomic era has seen remarkable advancement in our understanding of the molecular basis for disease, yet translation of basic discoveries into new disease treatments has arguably lagged behind. Recently, breakthroughs in genome editing technologies have created hope for their potential to directly treat the genetic causes of disease. Like any therapeutic intervention, genome editing should be considered in light of its potential risks and benefits. In this review, we highlight the promise of geno...

Nanoparticles for Nucleic Acid Delivery: Applications in Cancer Immunotherapy.

Immunotherapy has recently emerged as a powerful tool for cancer treatment. Early clinical successes from cancer immunotherapy have led to a growing list of FDA approvals, and many new therapies are in clinical and preclinical development. Nucleic acid therapeutics, including DNA, mRNA, and genome editing systems, hold significant potential as a form of immunotherapy due to its robust uses in cancer vaccines, adoptive T-cell therapies, and gene regulation towards the goal of treating cancer. However, these ...

One Prime for All Editing.

Many targeted base transversions, insertions, and deletions remain challenging due to the lack of precise and efficient genome editing technologies. Recently, Anzalone et al. reported a versatile approach to achieve all types of genome edits, shedding new light on correcting most genetic variants associated with diseases.

A role for alternative end-joining factors in homologous recombination and genome editing in Chinese hamster ovary cells.

CRISPR technologies greatly foster genome editing in mammalian cells through site-directed DNA double strand breaks (DSBs). However, precise editing outcomes, as mediated by homologous recombination (HR) repair, are typically infrequent and outnumbered by undesired genome alterations. By using knockdown and overexpression studies in Chinese hamster ovary (CHO) cells as well as characterizing repaired DNA junctions, we found that efficient HR-mediated genome editing depends on alternative end-joining (alt-EJ...

Off-target Editing by CRISPR-guided DNA base editors.

Base editing is a genome editing strategy that induces specific single nucleotide changes within genomic DNA. Two major DNA base editors, Cytosine base editors (CBEs) and Adenine base editors (ABEs), have been developed that consist of a Cas9 protein linked to a deaminase enzyme that catalyzes targeted base conversion directed by a sgRNA. This strategy has been used widely for precise genome editing because, unlike CRISPR-Cas nuclease-based genome editing systems, this strategy does not create double strand...

State-of-the-art CRISPR/Cas9 Technology for Genome Editing in Trypanosomatids.

CRISPR/Cas9 technology has revolutionized biology. This prokaryotic defense system against foreign DNA has been repurposed for genome editing in a broad range of cell tissues and organisms. Trypanosomatids are flagellated protozoa belonging to the order Kinetoplastida. Some of its most representative members cause important human diseases affecting millions of people worldwide, such as Chagas disease, sleeping sickness and different forms of leishmaniases. Trypanosomatid infections represent an enormous bur...

Precise in vivo genome editing via single homology arm donor mediated intron-targeting gene integration for genetic disease correction.

In vivo genome editing represents a powerful strategy for both understanding basic biology and treating inherited diseases. However, it remains a challenge to develop universal and efficient in vivo genome-editing tools for tissues that comprise diverse cell types in either a dividing or non-dividing state. Here, we describe a versatile in vivo gene knock-in methodology that enables the targeting of a broad range of mutations and cell types through the insertion of a minigene at an intron of the target gene...

Base Editing in Crops: Current Advances, Limitations, and Future Implications.

Targeted mutagenesis via genome editing technologies holds great promise in developing improved crop varieties to meet future demands. Point mutations or single nucleotide polymorphisms (SNPs) often determine important agronomic traits of crops. Genome editing-based single-base changes could generate elite trait variants in crop plants which help in accelerating crop improvement. Among the genome editing technologies, base editing has emerged as a novel and efficient genome editing approach which enables di...

Engineered Cas9 variant tools expand targeting scope of genome and base editing in rice.

Recently, some newly engineered SpCas9 variants, such as xCas9 3.7 and Cas9-NG, expand the PAM recognition site to NG and show function efficiency in mammalian cells. However, there is still not enough comparative data for their efficiencies on the genome and base editing in plants. In addition, there is also a lack of enhanced specific Cas9 variant with NG PAM. Here, we developed a serial of genome editing tools using different Cas9 variants containing the xCas9, the Cas9-NG and the enhanced specific Cas9-...

Anti-CRISPR AcrIIA5 Potently Inhibits All Cas9 Homologs Used for Genome Editing.

CRISPR-Cas9 systems provide powerful tools for genome editing. However, optimal employment of this technology will require control of Cas9 activity so that the timing, tissue specificity, and accuracy of editing may be precisely modulated. Anti-CRISPR proteins, which are small, naturally occurring inhibitors of CRISPR-Cas systems, are well suited for this purpose. A number of anti-CRISPR proteins have been shown to potently inhibit subgroups of CRISPR-Cas9 systems, but their maximal inhibitory activity is g...

Search-and-replace genome editing without double-strand breaks or donor DNA.

Most genetic variants that contribute to disease are challenging to correct efficiently and without excess byproducts. Here we describe prime editing, a versatile and precise genome editing method that directly writes new genetic information into a specified DNA site using a catalytically impaired Cas9 fused to an engineered reverse transcriptase, programmed with a prime editing guide RNA (pegRNA) that both specifies the target site and encodes the desired edit. We performed more than 175 edits in human cel...

Rational designs of in vivo CRISPR-Cas delivery systems.

The CRISPR-Cas system initiated a revolution in genome editing when it was, for the first time, demonstrated success in the mammalian cells. Today, scientists are able to readily edit the genome, regulate gene transcription, engineer posttranscriptional events, and image nucleic acids using CRISPR-Cas-based tools. However, to efficiently transport CRISPR-Cas into target tissues/cells remains challenging due to many extra- and intra-cellular barriers, therefore largely limiting the applications of CRISPR-bas...

Improving Editing Efficiency for the Sequences with NGH PAM Using xCas9-Derived Base Editors.

The development of CRISPR/Cas9-mediated base editors (BEs) provided a versatile tool for precise genome editing. The recently developed xCas9-derived base editors (xBEs) that recognize the NG PAM substantially expand the targeting scope in the genome, while their editing efficiency needs to be improved. Here, we described an improved version of xBEs by fusing the BPNLS and Gam to the N terminus of xBEs (BPNLS-Gam-xBE3 and BPNLS-xABE), and this version of base editor displayed higher targeting efficiency for...

Nanopore sequencing reveals a structural alteration of mirror-image duplicated genes in a genome-editing mouse line.

CRISPR-Cas9 technology has been used in various studies; however, it has also been found to introduce unexpected structural alternations. In this study, we used nanopore sequencing to characterize an unexpected structural alteration of mirror-image duplicated genes in a mouse line, in which we aimed to delete a part of the duplicated genes using genome editing. We removed low-molecular-weight DNA fragments and increased the input, which led to improved sequence performance. With 14.9 Gb input for whole-geno...

Therapeutic potential of CRISPR/Cas9 gene editing in engineered T-cell therapy.

Cancer patients have been treated with various types of therapies, including conventional strategies like chemo-, radio-, and targeted therapy, as well as immunotherapy like checkpoint inhibitors, vaccine and cell therapy etc. Among the therapeutic alternatives, T-cell therapy like CAR-T (Chimeric Antigen Receptor Engineered T cell) and TCR-T (T Cell Receptor Engineered T cell), has emerged as the most promising therapeutics due to its impressive clinical efficacy. However, there are many challenges and obs...

Rapid and efficient production of genome-edited animals by electroporation into oocytes injected with frozen or freeze-dried sperm.

Sperm preservation is a useful technique for maintaining valuable animal strains. Rat sperm could be frozen or freeze-dried in a simple Tris-EDTA solution (TE buffer), and oocytes that were fertilized with these sperm by intracytoplasmic sperm injection (ICSI) developed into offspring. Genome editing with the clustered regularly interspaced short palindromic repeat (CRISPR) and CRISPR-associated protein 9 (Cas9) system enables the rapid production of genetically modified rats. The recent innovative method, ...

Efficient Generation of Pathogenic A-to-G Mutations in Human Tripronuclear Embryos via ABE-Mediated Base Editing.

Base editing systems show their power in modeling and correcting the pathogenic mutations of genetic diseases. Previous studies have already demonstrated the editing efficiency of BE3-mediated C-to-T conversion in human embryos. However, the precision and efficiency of a recently developed adenine base editor (ABE), which converts A-to-G editing in human embryos, remain to be addressed. Here we selected reported pathogenic mutations to characterize the ABE in human tripronuclear embryos. We found effective ...

CRISPR/Cas9-Mediated Targeted Mutagenesis in Wheat Doubled Haploids.

CRISPR/Cas9-based genome editing technology has the potential to revolutionize agriculture, but many plant species and/or genotypes are recalcitrant to conventional transformation methods. Additionally, the long generation time of crop plants poses a significant obstacle to effective application of gene editing technology, as it takes a long time to produce modified homozygous genotypes. The haploid single-celled microspores are an attractive target for gene editing experiments, as they enable generation of...

A CRISPR/Cas9-based genome editing system for Rhodococcus ruber TH.

Rhodococcus spp. are organic solvent-tolerant strains with strong adaptive abilities and diverse metabolic activities, and are therefore widely utilized in bioconversion, biosynthesis and bioremediation. However, due to the high GC-content of the genome (∼70%), together with low transformation and recombination efficiency, the efficient genome editing of Rhodococcus remains challenging. In this study, we report for the first time the successful establishment of a CRISPR/Cas9-based genome editing system fo...

Engineering plants virus resistance: from RNA silencing to genome editing strategies.

Viral diseases severely affect crop yield and quality, thereby threatening global food security. Genetic improvement of plant virus resistance is essential for sustainable agriculture. In the last decades, several modern technologies were applied in plant antiviral engineering. Here we summarized breakthroughs of the two major antiviral strategies, RNA silencing and genome editing. RNA silencing strategy has been used in antiviral breeding for more than thirty years, and many crops engineered to stably expr...

The next generation of CRISPR-Cas technologies and applications.

The prokaryote-derived CRISPR-Cas genome editing systems have transformed our ability to manipulate, detect, image and annotate specific DNA and RNA sequences in living cells of diverse species. The ease of use and robustness of this technology have revolutionized genome editing for research ranging from fundamental science to translational medicine. Initial successes have inspired efforts to discover new systems for targeting and manipulating nucleic acids, including those from Cas9, Cas12, Cascade and Cas...

CRISPR/Cas: An intriguing genomic editing tool with prospects in treating neurodegenerative diseases.

The CRISPR/Cas genome editing tool has led to a revolution in biological research. Its ability to target multiple genomic loci simultaneously allows its application in gene function and genomic manipulation studies. Its involvement in the sequence specific gene editing in different backgrounds has changed the scenario of treating genetic disease. By unravelling the mysteries behind complex neuronal circuits, it not the only paved way in understanding the pathogenesis of the disease but helped in the develop...


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