All Gene Therapy articles – Page 5
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ArticleOvercoming operational challenges in CGT manufacturing with AI
In this Q&A, Dalip Sethi, scientific lead for Terumo Blood and Cell Technologies, Cell Therapy Technologies and Innovation portfolio, discusses the integration of AI into CGT manufacturing processes to enhance operational efficiency and accelerate treatment access. He also elucidates the strategies that are being considered to overcome hurdles when implementing ...
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ArticleThe rising demand for lentiviral vectors for in vivo gene therapy
Fuelled by advances in rare disease treatments and vaccination efforts, Natalia Elizalde, CBO at VIVEbiotech, discusses how the market demand for lentiviral vectors is evolving, the new therapeutic areas emerging as potential targets for in vivo gene therapy and the latest technological advancements in the development and delivery of in ...
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NewsSpinal muscular atrophy: searching for a cure
Organoids with SMA-pathology uncovered key findings about the disease, which could be utilised to develop new therapeutic options.
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NewsPrime editing corrects the CFTR gene mutation
The approach precisely and durably corrects the CFTR mutation in human lung cells, which could lead to superior treatments.
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NewsNovel version of CRISPR gene-editing protein engineered
Researchers have developed a new, compact EbCas12a variant that can be packaged into an all-in-one AAV system with its crRNA.
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ArticleEnhancing gene therapy with Circio
In this Q&A, Erik Wiklund, CEO of Circio, explains the key findings of their circVec circular RNA platform technology, why they chose AAV-based gene therapy for AATD as the lead programme, and their plans for the future to enhance the potency and reduce the cost of current gold-standard gene therapy. ...
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NewsGene editing for HSV-1 shows significant reduction of viral load
The experimental therapy eliminated 90 percent of HSV-1 after facial infection and 97 percent of HSV-1 after genital infection.
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ReportBeyond the lab: cell and gene therapy
Download this report now to access exclusive content on innovative cancer treatments, regenerative medicine, gene therapy, and expert insights from leading professionals in the field.
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NewsInvestigating new treatments for polycystic kidney disease
Organoid models enabled the researchers to study the effectiveness of eukaryotic ribosomal selective glycoside drugs on PKD cyst formation.
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NewsEvaluating novel gene therapies in a whole human liver
Use of the whole liver could revolutionise the development of viral vectors, providing more effective treatments for inherited diseases.
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How fusion proteins drive paediatric cancer
Researchers discover that fusion proteins and a gene regulatory protein complex interact through disordered domains.
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ArticleWomen in Stem with Dr Amber D. Van Laar
Dr Amber Van Laar shares her remarkable journey in STEM. From a childhood fascination with science and medicine to a pivotal role as VP Clinical Development, AskBio. In this interview, she explores the profound impact of her early exposure to neuro-oncology, the challenges faced as a physician-scientist, and the pursuit ...
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ArticleGamma delta T cells: a rising star in cancer therapy
Gamma delta (γδ) T cells comprise a distinct and powerful subpopulation of T cells, bridging innate and adaptive immunity, that holds great promise for treating cancer. IN8Bio co-founder and CEO William Ho and COO Kate Rochlin discuss what makes γδ T cells unique, how preclinical research suggests their antitumour activity ...
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ArticleA promising new option for male contraception
Scientists have targeted the SMRT-retinoic acid receptor interaction, which could provide a reversible and non-hormonal male contraceptive method.
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NewsCombatting HIV using gene therapy
Researchers have created a nanomedicine loaded with siRNAs, which demonstrated a 73 percent reduction in HIV replication.
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ArticleWomen in Stem with Dr Beate Mueller-Tiemann
Introducing Dr Beate Mueller-Tiemann, whose journey spans continents and disciplines, with a passion for biochemistry and molecular biology since her high school days. From elucidating pivotal proteins in cancer to monoclonal antibody discovery, Dr Mueller-Tiemann's contributions have not only transformed our understanding of diseases but have also fuelled research in ...
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NewsReplacing the PKP2 gene prolongs survival in ARVC patients
The new study suggests that this gene therapy method may combat ARVC in both early and more advanced stages of the condition.
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NewsOff-switches developed for CRISPR-Cas3 technologies
AcrlC8 and AcrlC9 prevent the CRISPR-Cas3 machine from binding to its DNA target site, providing a safer way to engineer the genome.
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WhitepaperArticle: Tips and Tricks for Leveraging Advanced Flow Cytometry
Tips and tricks for fully leveraging Advanced Flow Cytometry.
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ArticleThe science of ageing and restoring healthspan
Contrary to popular belief, ageing is not caused by just random wear and tear of our bodies over time but is instead caused by a discrete set of biological mechanisms that we now better understand and can target with therapies. Life Biosciences are specifically focused on restoring and prolonging one’s ...