PubMed Journals Articles About "Gene Editing Revolutionizing Medicine Causing Government Ethics Nightmare" RSS

18:11 EST 12th December 2018 | BioPortfolio

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Showing "Gene Editing Revolutionizing Medicine Causing Government Ethics Nightmare" PubMed Articles 1–25 of 28,000+

Optimization of sand fly embryo microinjection for gene editing by CRISPR/Cas9.

Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/Cas9 technology has rapidly emerged as a very effective tool for gene editing. Although great advances on gene editing in the medical entomology field have arisen, no attempts of gene editing have been reported in sand flies, the vectors of Leishmaniasis.

CRISPR-Cas9-mediated gene editing in human MPS I fibroblasts.

Mucopolysaccharidosis type I (MPS I) is a lysosomal storage disorder (LSD). It is caused by mutations in the IDUA gene, which lead to the accumulation of the glycosaminoglycans dermatan and heparan sulfate. The CRISPR-Cas9 system is a new and powerful tool that allows gene editing at precise points of the genome, resulting in gene correction through the introduction and genomic integration of a wildtype sequence. In this study, we used the CRISPR-Cas9 genome editing technology to correct in vitro the most c...

CRISPR/Cas9-mediated genome editing induces gene knockdown by altering the pre-mRNA splicing in mice.

Clustered Regularly Interspaced Short Palindromic Repeats/CRISPR associated protein 9 (CRISPR/Cas9) has been wildly used to generate gene knockout models through inducing indels causing frame-shift. However, there are few studies concerning the post-transcript effects caused by CRISPR-mediated genome editing.

In vitro-transcribed guide RNAs trigger an innate immune response via the RIG-I pathway.

Clustered, regularly interspaced, short palindromic repeat (CRISPR)-CRISPR-associated 9 (Cas9) genome editing is revolutionizing fundamental research and has great potential for the treatment of many diseases. While editing of immortalized cell lines has become relatively easy, editing of therapeutically relevant primary cells and tissues can remain challenging. One recent advancement is the delivery of a Cas9 protein and an in vitro-transcribed (IVT) guide RNA (gRNA) as a precomplexed ribonucleoprotein (RN...

Targeted Gene Knock Out Using Nuclease-Assisted Vector Integration: Hemi- and Homozygous Deletion of JAG1.

Gene editing technologies are revolutionizing fields such as biomedicine and biotechnology by providing a simple means to manipulate the genetic makeup of essentially any organism. Gene editing tools function by introducing double-stranded breaks at targeted sites within the genome, which the host cells repair preferentially by Non-Homologous End Joining. While the technologies to introduce double-stranded breaks have been extensively optimized, this progress has not been matched by the development of metho...

Family Medicine Ethics: An Integrative Approach.

The practice of modern medical ethics is largely acute, episodic, fragmented, problem-focused, and institution-centered. Family medicine, in contrast, is built upon a relationship-based model of care that is accessible, comprehensive, continuous, contextual, community-focused and patient-centered. "Doing ethics" in the day-to-day practice of family medicine is therefore different from doing ethics in many other fields of medicine, emphasizing different strengths and exemplifying different values. For family...

Unleashing the Therapeutic Potential of CAR-T Cell Therapy Using Gene-Editing Technologies.

Chimeric antigen receptor (CAR) T-cell therapy, an emerging immunotherapy, has demonstrated promising clinical results in hematological malignancies including B-cell malignancies. However, accessibility to this transformative medicine is highly limited due to the complex process of manufacturing, limited options for target antigens, and insufficient anti-tumor responses against solid tumors. Advances in gene-editing technologies, such as the development of Zinc Finger Nucleases (ZFNs), Transcription Activat...

Sleep spindles are altered in early- but not late-onset nightmare recallers.

Nightmares are a common sleep disorder, defined as highly disturbing mentation which usually awakens the individual from rapid eye movement (REM) sleep. While nightmares are mainly a REM sleep phenomenon, Picard-Deland et al., (2017) recently showed an association between nightmare recall and sleep spindles, which are a non-rapid eye movement (NREM) oscillatory feature. Their results pointed to fewer slow spindles and a higher oscillatory frequency for fast spindles among frequent nightmare recallers compa...

65 YEARS OF THE DOUBLE HELIX: The advancements of gene editing and potential application to hereditary cancer.

Hereditary cancer predisposition syndromes are associated with germline mutations that lead to increased vulnerability for an individual to develop cancers. Such germline mutations in tumour suppressor genes, oncogenes and genes encoding for proteins essential in DNA repair pathways and cell cycle control can cause overall chromosomal instability in the genome and increase risk in developing cancers. Gene correction of these germline mutations to restore normal protein functions is anticipated as a new ther...

Extension of the crRNA enhances Cpf1 gene editing in vitro and in vivo.

Engineering of the Cpf1 crRNA has the potential to enhance its gene editing efficiency and non-viral delivery to cells. Here, we demonstrate that extending the length of its crRNA at the 5' end can enhance the gene editing efficiency of Cpf1 both in cells and in vivo. Extending the 5' end of the crRNA enhances the gene editing efficiency of the Cpf1 RNP to induce non-homologous end-joining and homology-directed repair using electroporation in cells. Additionally, chemical modifications on the extended 5' en...

Novel Gene-Editing Technique Cures β-Thalassemia in Utero: A novel peptide nucleic acid-based gene-editing technique using a nanoparticle delivery system seemingly cured beta thalassemia in fetal mice.

Justice in CRISPR/Cas9 Research and Clinical Applications.

CRISPR/Cas9 is a rapidly developing gene editing technology that will soon have many clinical applications. As with many other new technologies, somatic gene editing with CRISPR/Cas9 raises concerns about equitable access to therapies by historically disenfranchised racial and ethnic minorities. We describe justice concerns related to CRISPR/Cas9, including its potential impact on historically mistreated populations through underrepresentation of minorities in genomic databases and the potential for dispara...

Cytogenetic Analysis of the Results of Genome Editing on the Cell Model of Parkinson's Disease.

We performed a cytogenetic analysis of the results of CRISPR/Cas9-correction of G2019S mutation in LRRK2 gene associated with Parkinson's disease. Genome editing was performed on induced pluripotent stem cells derived from fibroblasts of a patient carrying this mutation. A mosaic variant of tetraploidy 92 XXYY/46,XY (24-43% cells from various clones) was found in neuronal precursors differentiated from the induced pluripotent stem cells after gene editing procedure. Solitary cases of translocations and chro...

Off-target predictions in CRISPR-Cas9 gene editing using deep learning.

The prediction of off-target mutations in CRISPR-Cas9 is a hot topic due to its relevance to gene editing research. Existing prediction methods have been developed; however, most of them just calculated scores based on mismatches to the guide sequence in CRISPR-Cas9. Therefore, the existing prediction methods are unable to scale and improve their performance with the rapid expansion of experimental data in CRISPR-Cas9. Moreover, the existing methods still cannot satisfy enough precision in off-target predic...

Epigenome Editing Enters the Arena: A New Tool to Reveal (and Reverse?) Pathologic Gene Regulation.

CRISPR-Cpf1-mediated genome editing and gene regulation in human cells.

Clustered regularly interspaced short palindromic repeat (CRISPR) system is being championed as a robust and flexible tool for genome editing. Compared with CRISPR associated protein 9 (Cas9), the CRISPR from Prevotella and Francisella 1 (Cpf1) protein has some distinct characteristics, including RNase activity, T-rich protospacer adjacent motif (PAM) preference and generation of sticky cutting ends. The extremely low propensity of off-target effects and relatively high editing efficiency represent prominen...

Single-nucleotide variants in human RNA: RNA editing and beyond.

Through analysis of paired high-throughput DNA-Seq and RNA-Seq data, researchers quickly recognized that RNA-Seq can be used for more than just gene expression quantification. The alternative applications of RNA-Seq data are abundant, and we are particularly interested in its usefulness for detecting single-nucleotide variants, which arise from RNA editing, genomic variants and other RNA modifications. A stunning discovery made from RNA-Seq analyses is the unexpectedly high prevalence of RNA-editing events,...

Allele-specific genome editing using CRISPR-Cas9 is associated with loss of heterozygosity in diploid yeast.

Targeted DNA double-strand breaks (DSBs) with CRISPR-Cas9 have revolutionized genetic modification by enabling efficient genome editing in a broad range of eukaryotic systems. Accurate gene editing is possible with near-perfect efficiency in haploid or (predominantly) homozygous genomes. However, genomes exhibiting polyploidy and/or high degrees of heterozygosity are less amenable to genetic modification. Here, we report an up to 99-fold lower gene editing efficiency when editing individual heterozygous loc...

Addressing Medical Students' Negative Bias Toward Patients With Obesity Through Ethics Education.

Negative bias toward patients with obesity is an ethical challenge in patient care. Several interventions to mitigate medical students' negative weight bias have been tried but none with an explicit focus on ethics. Here we describe first-year medical students' attitudes toward obesity and our effort to improve their attitudes through an innovative ethics session embedded within the required course, "Obesity, Nutrition, and Behavior Change," at Johns Hopkins University School of Medicine.

CRISPR/Cas: technique to repair DNA errors: is a clinical breakthrough near?

CRISPR/Cas gene editing makes it much easier to make targeted changes in the DNA of human cells than other forms of gene therapy. This revolutionary technology offers spectacular opportunities to study gene functions; the clinical consequences of gene variations in patients can be determined much faster. The efficacy and accuracy of CRISPR/Cas is so impressive that a breakthrough to therapeutic applications is approaching fast. CRISPR/Cas is already being used in immunotherapy against cancer, and trials for...

Drug Addiction: Mechanisms of Nicotine Dependence Unmasked by Gene Editing.

New gene editing technologies are enabling exploration of previously intractable features of genetic risk for drug addiction. A recent study using this technology reveals new insights into how a mutation linked to tobacco dependence influences the addictive properties of nicotine.

On the necessity of philosophy in medical education.

Today, the study and practice of medicine are treated with a natural scientific approach. However, medical action has its foundations not only in the natural sciences and technology, but also in ethics, humanity and philosophy. The Wuerzburg Philosophicum teaches, apart from medical ethics, a systematic analysis of a theory of medicine in the context of other sciences, the theory of knowledge, anthropology and hermeneutics.

Progress in the application of CRISPR: From gene to base editing.

The system of clustered regularly interspaced short palindromic repeats (CRISPR) and CRISPR-associated endonucleases (Cas) has been utilized for genome editing with great accuracy and high efficiency in generating gene knockout, knockin, and point mutations in eukaryotic genomes. However, traditional CRISPR/Cas9 technology introduces double-stranded DNA breaks (DSBs) at a target locus as the first step to make gene corrections, which easily results in undesired mutations. Thus, it is necessary to develop n...

A Crispr Future for Gene-Editing Regulation: a Proposal for an Updated Biotechnology Regulatory System in an Era of Human Genomic Editing.

Recent developments in gene-editing technology have enabled scientists to manipulate the human genome in unprecedented ways. One technology in particular, Clustered Regularly Interspaced Short Pallindromic Repeat (CRISPR), has made gene editing more precise and cost-effective than ever before. Indeed, scientists have already shown that CRISPR can eliminate genes linked to life-threatening diseases from an individual's genetic makeup and, when used on human embryos, CRISPR has the potential to permanently el...

Effective PEI-mediated delivery of CRISPR-Cas9 complex for targeted gene therapy.

The-state-of-art CRISPR/Cas9 is one of the most powerful among the approaches being developed to rescue fundamental causes of gene-based inheritable diseases. Several strategies for delivering such genome editing materials have been developed, but the safety, efficacy over time, cost of production, and gene size limitations are still under debate and must be addressed to further improve applications. In this study, we evaluated branched forms of the polyethylenimine (PEI)-branched PEI 25 kDa (BPEI-25K)-and ...

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