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Drug Target Review – Issue 2 2023
A new Drug Target Review issue is now ready to download! This issue features articles on cell and gene therapy, imaging and screening.
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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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ebook: Breaking Barriers in Cell Engineering
Download this FREE ebook now and explore single-cell analysis, unlocking the secrets of therapeutic efficacy and safety. Immerse yourself in exclusive content, including cutting-edge cell line development articles and riveting interviews with industry experts. Don't miss your chance to discover the future of cellular therapies – start your journey today!
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Nerve regeneration offers hope for blindness and paralysis
US researchers uncover that certain nerve cells that have the capacity to regrow, are necessary for axon regeneration, providing hope for conditions like blindness and paralysis.
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New drug boosts foetal haemoglobin to fight sickle cell disease
Researchers are exploring the potential use of HDAC inhibitors, a group of drugs known for their capability to impede tumour cell division, to reduce the pain and damage associated with sickle cell disease. The drugs have shown initial evidence of reactivating the gene responsible for producing foetal haemoglobin, which cannot…
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USC researchers zoom into human genome with mammal DNA
USC researcher Dr Steven Gazal and his team have identified human genome base pairs that have remained constant over millions of years of mammalian evolution. These base pairs are linked to human disease. Using DNA from 240 mammal species, they identified genetic variations affecting an individual's survival and reproduction.
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New insight into BRCA2 gene mutations and cancer risk
A recent study from researchers at the University of California examines the BRCA2 gene, which plays a crucial role in repairing damaged DNA, and possible links with increased risk of breast and ovarian cancer.
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Chances of eliminating HIV infection increased by dual gene-editing method
US researchers suggest that combing a dual gene-editing approach with antiretroviral drugs can eliminate HIV infections in animal models.
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Scientists uncover new insights into rare childhood liver cancer
Researchers from the Netherlands have utilised organoids and the CRISPR-Cas9 "molecular scissor" system to better understand the features and biology of fibrolamellar carcinoma (FLC), a rare type of liver cancer that affects adolescents and young adults.
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Inflammation ‘brake’ gene may impact kidney disease outcomes
Australian researchers uncover a gene that controls inflammation in kidney disease, which could pave the way for more precise disease diagnostics and personalised treatments.
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Recombining drugs helps restore gene’s ability to signal for death of leukaemia cells
US researchers find combination of chloroquine and venetoclax promotes cancer cell death in mouse models with acute myeloid leukaemia (AML).
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Combination therapy prevails against BET inhibitor resistance
St. Jude Children's Research Hospital, US, scientists created a new combination therapy method to tackle drug resistance in a type of leukaemia with KMT2A gene rearrangement.
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Step forward in gene therapy to treat cause of sudden cardiac arrest
Using gene therapy, the researchers corrected abnormal heart rhythms by restoring a protein that heart cells need to establish connections with one another.
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Research shows lipid nanoparticles are highly effective in gene therapy
Lipid nanoparticles have been used to encapsulate CRISPR-Cas9 and deliver it to cells in mice, where it was highly effective at knocking down expression of a target protein.
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Usher syndrome: first nonhuman primate model
US researchers evaluate an experimental gene therapy in the first ever nonhuman primate model for Usher Syndrome.
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Therapeutic strategy using p53 induces cancer cell death
US researchers spotlight how p53, the most frequently mutated tumour suppressor gene, can be activated against cancer cells.
