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Nano-micelles used in CRISPR genome editing of mouse brains
Researchers have reported that nano-micelles can be used to efficiently deliver CRISPR-Cas9 to edit genes in the brains of mice.
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Gene Therapy
Gene Therapy is the introduction of normal genes into cells in place of missing or defective ones in order to correct genetic disorders.
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CRISPR-dead Cas9 could offer non-addictive treatment for pain, study shows
A new CRISPR gene therapy for chronic pain has been shown in mice to temporarily repress a gene involved in sensing pain.
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Machine learning used to generate AAV capsids for gene therapy
A study has used artificial intelligence to reveal adeno-associated virus (AAV) capsid variants for use in gene therapies.
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TALEN is a more efficient gene editing tool for tightly packed DNA, finds study
TALEN was shown to be almost five times more efficient than CRISPR-Cas9 at locating and editing genes in heterochromatin.
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Hyper-interleukin-6 allows paralysed mice to walk again
Treating only a few nerve cells with the hyper-interleukin-6 (hIL-6) gene therapy stimulated the regeneration of nerves.
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Mitochondrial replacement therapy (MRT) shown to be safe in monkeys
A long-term study of macaques given mitochondrial replacement therapy (MRT) found that both treated individuals and their offspring were healthy and developed normally.
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Tweaking synonymous sites for gene therapy and vaccines
Professor Laurence D Hurst explains why understanding the nucleotide mutations in viruses, including SARS-CoV-2, can have significant implications for vaccine design.
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New gene therapy could treat dominant optic atrophy
Researchers have found that an OPA1-targeted gene therapy can treat dominant optic atrophy in pre-clinical trials.
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CRISPR: a molecular scissor set to change the world
In October this year, Emmanuelle Charpentier and Jennifer Doudna, the two scientists who pioneered the revolutionary gene-editing technology CRISPR, were awarded the Nobel Prize in Chemistry. Here, Pushpanathan Muthuirulan discusses the potential for this technology and the importance of using it safely, ethically and responsibly.
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‘Neuroregenerative’ study uses glial cells for stroke repair in primates
In rhesus macaques, a team were able to induce neural regeneration from brain internal glial cells, repairing damage from stroke.
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Expert view: ddPCR whole cell DNA workflow efficiently measures success of CAR T-cell production
Not every step of CAR T-cell manufacturing can be tightly regulated, introducing some significant quality control challenges for companies developing CAR T cancer therapies
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Defusing antibody neutralisers in AAV gene therapy
Adeno-associated virus (AAV)-mediated gene therapies allow for the treatment of a growing number of diseases; however, the presence of neutralising antibodies can lead to limitations of this technology, particularly for patients who may be excluded due to these pre-existing or developing neutralising antibodies. Recently, a study was published in Nature…
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Antibodies In-Depth Focus 2020
Included in this in-depth focus are articles on antibody therapeutics to treat COVID-19, how AAV antibodies can be neutralised, the role of antibodies in cancer therapy and bispecific antibodies for immuno-oncology drugs.
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Drug Target Review – Issue 3 2020
This issue includes articles that explore how a next-generation genomics platform can be used for COVID-19 research, the elimination of neutralising AAV antibodies for gene therapies and a new quick and cost-effective biomarker technology for cancer diagnostics. Also in this issue are features on antibody therapeutics for COVID-19 and targets…
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New CRISPR system edits body’s response to prevent immunity against AAVs
A novel CRISPR system that suppresses genes related to adeno-associated virus (AAV) antibody production has been developed to prevent immunity against the gene therapy.