Gene Therapy News and Research

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Gene therapy is an experimental technique that uses genes to treat or prevent disease. In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patient’s cells instead of using drugs or surgery.

Study: R-spondin growth factor reduces intestinal tumors in early phase of colorectal cancer

Colorectal cancer constitute the third most common cancer in the Western world. Their incidence constantly increases as the population ages, and despite recent advances in colorectal cancer therapy, the cancer metastasizes in every third of colorectal cancer patients, and a significant part of these patients eventually die from the disease

24 Sep 2020

Study finds spectrum of genes responsible for inherited hearing loss in Israeli Jewish families

Until now, seven genes were known to be involved in hearing loss in Israel's Jewish population. A new study led by Zippora Brownstein, PhD, and Prof. Karen Avraham from the Sackler Faculty of Medicine at Tel Aviv University has found that 32 genes are responsible for inherited hearing loss in Israeli Jewish families.

23 Sep 2020

OXGENE launches a scalable, plasmid-free manufacturing system for AAV

OXGENE™, a biotechnology company designing and developing scalable gene therapy technologies, has today announced the launch of its scalable, plasmid-free manufacturing system for AAV.

From OXGENE 23 Sep 2020

Researchers generate accurate replications of human retinas in a dish

Scientists have generated accurate replications of human retinas in culture that can be used to pinpoint the specific types of cells affected by genetic eye diseases.

21 Sep 2020

Providing personalized care for bleeding disorders

An interview with Dr. Carmen Escuriola-Ettingshausen MD, Mörfelden-Walldorf, Germany on providing personalized care for bleeding disorders.

From Takeda 21 Sep 2020

Providing personalized care for bleeding disorders

An interview with Dr. Carmen Escuriola-Ettingshausen MD, Mörfelden-Walldorf, Germany on providing personalized care for bleeding disorders.

From Takeda 21 Sep 2020

Gene therapy used to overcome the cardiac effects of Freidreich’s ataxia

Gene therapy was successfully used to overcome the cardiac effects of Freidreich’s ataxia (FA) in a mouse model of the disease, as reported in the peer-reviewed journal Human Gene Therapy. Click here to read the full-text article free online through October 18, 2020.

19 Sep 2020

S-pseudotyped VLPs offer promising COVID-19 vaccine platform

A new study published on the preprint server bioRxiv* in September 2020 reports on a Moloney murine leukemia virus (MLV) platform that produces VLPs expressing the important spike antigen of severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2).

18 Sep 2020

Researchers compare developed carriers for delivery of genome editing tools with existing analogs

Researchers from Peter the Great St.Petersburg Polytechnic University in collaboration with colleagues from the Pavlov University, ITMO University, and the University of Hamburg compared their developed carriers for delivery of genome editing (GE) tools with other available analogs.

16 Sep 2020

UC Davis uses microneedles to deliver gene therapy for retinal diseases

A novel approach to delivering gene therapy for retinal diseases eliminates the need for complex eye surgery and treats more damaged cells than existing methods, a team of UC Davis physicians and veterinary eye specialists has found.

16 Sep 2020

RCas9 gene therapy clears toxic RNA buildup in mouse model of myotonic dystrophy

Myotonic dystrophy type I is the most common type of adult-onset muscular dystrophy. People with the condition inherit repeated DNA segments that lead to the toxic buildup of repetitive RNA, the messenger that carries a gene's recipe to the cell's protein-making machinery.

14 Sep 2020

Earlier treatment with steroids reduces adverse outcomes in patients with septic shock

Some critically ill patients with septic shock need medications called vasopressors to correct dangerously low blood pressure.

14 Sep 2020

Study may expedite the process to cure blindness and macular degeneration

While there is no cure for blindness and macular degeneration, scientists have accelerated the process to find a cure by visualizing the inner workings of the eye and its diseases at the cellular level.

11 Sep 2020

Consortium compiles recommendations for measuring HIV reservoir size in cure-directed studies

Spearheaded by scientists at The Wistar Institute, top worldwide HIV researchers from the BEAT-HIV Martin Delaney Collaboratory to Cure HIV-1 Infection by Combination Immunotherapy compiled the first comprehensive set of recommendations on how to best measure the size of persistent HIV reservoirs during cure-directed clinical studies.

7 Sep 2020

CRISPR-based system suppresses genes related to AAV antibody production

Gene therapy generally relies on viruses, such as adeno-associated virus (AAV), to deliver genes into a cell. In the case of CRISPR-based gene therapies, molecular scissors can then snip out a defective gene, add in a missing sequence or enact a temporary change in its expression, but the body's immune response to AAV can thwart the whole endeavor.

4 Sep 2020

AAV gene therapy often leads to dorsal root ganglion pathology, show studies

A meta-analysis of non-human primate (NHP) studies showed that adeno-associated virus (AAV) gene therapy often caused dorsal root ganglion (DRG) pathology.

3 Sep 2020

New Gyrolab AAVX Titer Kit launched to support the development cell and gene therapies

Gyros Protein Technologies AB, a pioneer in automated nanoliter-scale immunoassays and a leading provider of peptide synthesizers and reagents, today announced the introduction of Gyrolab® AAVX Titer Kit for adeno-associated virus (AAV) vector titer determination in AAV vector manufacturing.

From Gyros Protein Technologies AB 3 Sep 2020

Trinity College researchers to participate in gene-based clinical trial for motor neuron disease

Researchers at Trinity College Dublin are to participate in an early phase gene-based clinical trial for motor neuron disease (MND). The research team represents one of only four centres in Europe to take part in such an exciting clinical trial for this devastating disease.

2 Sep 2020

New microgel drug delivery method could improve effectiveness of pancreatic islet transplants

Pancreatic islet transplants, which revive insulin production to treat type 1 diabetes, only last an average of three years.

31 Aug 2020

A powerful tool for separation and sizing of virus mixtures

Postnova Analytics has published an applications report that presents data from a collaboration with the Analytical Development group at Biogen Inc. on separation of a virus mixture using Asymmetrical Flow Field-Flow Fractionation and measurement of their radius of gyration.

From Postnova Analytics 27 Aug 2020

Gene Therapy News and Research

Further Reading

Study: R-spondin growth factor reduces intestinal tumors in early phase of colorectal cancer

Study finds spectrum of genes responsible for inherited hearing loss in Israeli Jewish families

OXGENE launches a scalable, plasmid-free manufacturing system for AAV

Researchers generate accurate replications of human retinas in a dish

Providing personalized care for bleeding disorders

Providing personalized care for bleeding disorders

Gene therapy used to overcome the cardiac effects of Freidreich’s ataxia

S-pseudotyped VLPs offer promising COVID-19 vaccine platform

Researchers compare developed carriers for delivery of genome editing tools with existing analogs

UC Davis uses microneedles to deliver gene therapy for retinal diseases

RCas9 gene therapy clears toxic RNA buildup in mouse model of myotonic dystrophy

Earlier treatment with steroids reduces adverse outcomes in patients with septic shock

Study may expedite the process to cure blindness and macular degeneration

Consortium compiles recommendations for measuring HIV reservoir size in cure-directed studies

CRISPR-based system suppresses genes related to AAV antibody production

AAV gene therapy often leads to dorsal root ganglion pathology, show studies

New Gyrolab AAVX Titer Kit launched to support the development cell and gene therapies

Trinity College researchers to participate in gene-based clinical trial for motor neuron disease

New microgel drug delivery method could improve effectiveness of pancreatic islet transplants

A powerful tool for separation and sizing of virus mixtures

Pioneering Live Cell Imaging - Yokogawa's Impact and Innovations

Revolutionizing Life Science: An Interview with SCIEX on ASMS, the SCIEX 7500+ System, and AI-Driven Quantitation

From Discovery Biology to ELRIG Chair

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