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Gene Therapy is the introduction of normal genes into cells in place of missing or defective ones in order to correct genetic disorders.
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…
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.
Ophthalmology and engineering combine in a novel nanoparticle-delivered gene therapy approach to treating wet age-related macular degeneration.
Scientists created a technique to give photoreceptors infrared-light sensitivity, allowing the animal models to see infrared heat signatures.
Researchers reveal protospacer adjacent motif mutations (PAM sites) on the NRF2 gene of cancers could be used to guide CRISPR gene editing.
Novel promoters based on those in the herpes viruses, enabled delivery of larger genes and increased the period they were active in the nervous system.
Researchers have used high-throughput screening on AAV vector capsid libraries to identify which ones are best for certain gene therapies.
Researchers have shown a gene therapy to correct the genetic mutation that causes Danon disease is successful in pre-clinical trials.
Researchers have successfully treated cardiac dysfuntion in mice models of Barth syndrome by using a gene therapy to replace TAZ.
A study has shown that altering amino acid residues in Cas9 to produce new variants can produce a vector with increased gene editing specificity.
Researchers have developed a new regenerative gene therapy using neurogenic differentiation, which has shown efficacy treating Huntington's disease in mice.
Researchers have created a method to activate CAR T cells with blue LED light to successfully combat solid tumours in mice.
Results of an international collaboration show promise for the future of motor neurone disease treatment efforts, as protective gene is identified.
Researchers have developed a new CRISPR technique, using a minigene, which was inserted into mouse DNA, resulting in improved liver disease symptoms.
Mice with congenital blindness have shown significant improvement in vision after undergoing a new gene therapy.
