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Breakthrough gene therapy method utilizes bird retrotransposon for safe gene insertion

The recent approval of a CRISPR-Cas9 therapy for sickle cell disease demonstrates that gene editing tools can do a superb job knocking out genes to cure hereditary disease.

20 Feb 2024

CRISPR revolutionized: New method edits genes directly in the body

Most approved gene therapies today, including those involving CRISPR-Cas9, work their magic on cells removed from the body, after which the edited cells are returned to the patient.

1 Feb 2024

Asimov launches LV Edge Packaging System to optimize lentivirus production

New off-the-shelf system reduces the cost and risk of lentiviral (LV) production for cell and gene therapy developers.

From Asimov 29 Jan 2024

Penn analysis shows low risk of secondary cancers following CAR T cell therapy

The development of any type of second cancer following CAR T cell therapy is a rare occurrence, as found in an analysis of more than 400 patients treated at Penn Medicine, researchers from the Perelman School of Medicine at the University of Pennsylvania reported today in Nature Medicine.

24 Jan 2024

Breakthrough in gene editing: Enhanced virus-like particles promise new era in genetic disease treatment

Researchers developed an advanced prime editor-engineered virus-like particle (PE-eVLP) system, significantly enhancing gene editing efficiency in human cells and showing potential for treating genetic diseases.

9 Jan 2024

Revolutionary RNA-based switch offers new control over gene expression in mammalian cells

Researchers developed an RNA-based switch, the pA regulator system, to control gene expression in mammalian cells by modulating synthetic polyA signal cleavage, offering a novel approach for gene therapy applications.

4 Jan 2024

Texas Children's Hospital delivers first-ever gene therapy for Rett syndrome

Texas Children's Hospital, an internationally-recognized, top-ranked children's hospital and pediatric research center affiliated with Baylor College of Medicine, is the first to deliver a novel gene therapy to treat Rett syndrome in pediatric patients.

1 Dec 2023

Proof-of-concept study holds promise for safer gene therapy techniques for treating immunodeficiencies

Researchers described an approach to edit the recombination-activating gene 2 (RAG2) locus for therapeutic applications.

1 Nov 2023

Towards xenotransplantation: Genetically altered pig kidneys prove viable in non-human primates

Researchers used a genetically engineered porcine donor to develop kidney grafts.

11 Oct 2023

Study finds α5 integrin as a potential therapeutic target in amyotrophic lateral sclerosis

Researchers examined the therapeutic effects of targeting integrins for treating Amyotrophic lateral sclerosis (ALS).

3 Aug 2023

Robotic system may accelerate studies in diverse areas of C. elegans biology

A robotic system capable of imaging and transferring the model organism Caenorhabditis elegans could replace hours of tedious lab work, according to a recent study.

5 Jul 2023

Transgenic mouse model reveals surprising new connections between peripheral nerves and the heart

Oxidative stress – characterized by elevated levels of unstable molecules called reactive oxygen species– is associated with neurodegeneration and cardiovascular disease.

14 Jun 2023

Single dose of anti-Müllerian hormone gene therapy can induce long-term contraception in domestic cats

Currently there are no contraceptives capable of producing permanent sterilization in companion animals. Spaying, the surgical removal of the ovaries and uterus, is the most widely used strategy to control unwanted reproduction in female cats.

6 Jun 2023

Study explores the role of dynamin protein DYN-1 in regenerative axonal fusion

A study explores the role of the dynamin protein DYN-1 in axonal fusion. The axon is a long, thin protrusion of nerve cells that carries electrical impulses.

9 May 2023

Scientists uncover novel role of human-CIDEC gene in improving metabolic dysfunction and cardiovascular health

A team of researchers from Ohio University's Heritage College of Osteopathic Medicine recently discovered a novel role of human-CIDEC gene in improving metabolic dysfunction and cardiovascular health.

29 Mar 2023

Researchers optically evoke tachycardia enhanced anxiety-like behaviour

In a recent study published in the journal Nature, researchers devised a non-invasive optogenetic and wearable cardiac pacemaker for accurate and specific control of cardiac rhythm for ≤900 beats per minute (bpm), enabled by micro-light-emitting display (LED) harness and systemic channelrhodopsin delivery.

6 Mar 2023

Transgene News and Research

Breakthrough gene therapy method utilizes bird retrotransposon for safe gene insertion

CRISPR revolutionized: New method edits genes directly in the body

Asimov launches LV Edge Packaging System to optimize lentivirus production

Penn analysis shows low risk of secondary cancers following CAR T cell therapy

Breakthrough in gene editing: Enhanced virus-like particles promise new era in genetic disease treatment

Revolutionary RNA-based switch offers new control over gene expression in mammalian cells

Texas Children's Hospital delivers first-ever gene therapy for Rett syndrome

Proof-of-concept study holds promise for safer gene therapy techniques for treating immunodeficiencies

Towards xenotransplantation: Genetically altered pig kidneys prove viable in non-human primates

Study finds α5 integrin as a potential therapeutic target in amyotrophic lateral sclerosis

Robotic system may accelerate studies in diverse areas of C. elegans biology

Transgenic mouse model reveals surprising new connections between peripheral nerves and the heart

Single dose of anti-Müllerian hormone gene therapy can induce long-term contraception in domestic cats

Study explores the role of dynamin protein DYN-1 in regenerative axonal fusion

Scientists uncover novel role of human-CIDEC gene in improving metabolic dysfunction and cardiovascular health

Researchers optically evoke tachycardia enhanced anxiety-like behaviour

Genome editing could help save rice crops threatened by climate change

New family of adeno-associated viral vectors found to be better at delivering genetic cargo

New control system could be used to finely tune the production of useful proteins

Study finds telomeres can set damage thresholds for cancer cells above which they cannot continue to divide

Expanding research and clinical options for children with cancer

Examining how pain could play a direct protective role in the gut

From Puberty to Menopause: Clue’s CEO, Audrey Tsang on the Power of Femtech

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