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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Gene Therapy is the introduction of normal genes into cells in place of missing or defective ones in order to correct genetic disorders.
Professor Laurence D Hurst explains why understanding the nucleotide mutations in viruses, including SARS-CoV-2, can have significant implications for vaccine design.
Researchers have found that an OPA1-targeted gene therapy can treat dominant optic atrophy in pre-clinical trials.
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.
In rhesus macaques, a team were able to induce neural regeneration from brain internal glial cells, repairing damage from stroke.
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
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…
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.