List view / Grid view
Gene Therapy is the introduction of normal genes into cells in place of missing or defective ones in order to correct genetic disorders.
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…
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.
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.
