addgene Blog

JUNE 22, 2023

Viruses have become a regular part of basic biological research as well as clinical therapy. These biological tools are useful because they ’ re derived from viruses that can infect people, cells, and animals. Some of these viruses are completely inert, but others can cause diseases. Nervous about handling viruses? That ’ s okay!

Virus 97

Virus Therapies Disease Research 97

Broad Institute

JULY 19, 2023

Researchers reprogram gene therapy viral vectors to bind specific protein targets By Allessandra DiCorato July 19, 2023 Breadcrumb Home Researchers reprogram gene therapy viral vectors to bind specific protein targets A new screening method zeroes in on adeno-associated viruses that enter the brain through a defined mechanism.

Therapies 98

Therapies Research Engineering Virus 98

Broad Institute

JUNE 27, 2024

There are currently no treatments, but researchers from the Whitehead Institute for Biomedical Research and Broad Institute of MIT and Harvard have developed an approach that could one day be used to turn off the gene encoding this protein throughout the brain to treat or even prevent prion disease.

Disease 142

Disease Therapies Clinical Trials Engineering 142

SCIENMAG: Medicine & Health

JUNE 30, 2023

In a new study, the research team, which includes entomologists with the University of Florida, the Agricultural Research Service-USDA, Louisiana State University and […]

Virus 84

Virus Therapies Research 84

SCIENMAG: Medicine & Health

JUNE 20, 2023

EDMONTON — Combining a cancer-targeting virus with radiation to treat brain cancer in mice was more effective than either therapy on its own according to University of Alberta research, providing hope for new treatments that combine immunotherapy with traditional surgery, chemotherapy or radiation.

Virus 83

Virus Therapies Treatment Research 83

Broad Institute

JANUARY 8, 2024

Williams January 8, 2024 Credit: Susanna Hamilton, Broad Communications Researchers have developed virus-like particles that can deliver gene-editing cargo to cells, including those in the mouse brain. Researchers have used a variety of methods to deliver these molecular machines to cells, including lipid nanoparticles and viruses.

Engineering 144

Engineering Research Virus DNA 144

Broad Institute

AUGUST 7, 2024

Machine learning approach helps researchers design better gene-delivery vehicles for gene therapy By Allessandra DiCorato August 7, 2024 Breadcrumb Home Machine learning approach helps researchers design better gene-delivery vehicles for gene therapy The method could accelerate the development of more effective adeno-associated viruses (AAVs).

Therapies 84

Therapies Research Engineering Packaging 84

Drug Target Review

OCTOBER 2, 2024

Cell and gene therapies (CGTs) have made significant advancements in treating oncological diseases, with therapies like CAR-T cell treatments transforming cancer care. However, cell-based therapies are intended to leverage those healthy cells via transplant to regenerate damaged tissue.

Therapies 98

Therapies Disease Engineering RNA 98

Drug Target Review

JULY 5, 2023

Induced pluripotent stem cell (iPSC) therapies have the potential to treat a wide variety of devastating diseases. While iPSC therapies hold great promise, several challenges remain, including poor infiltration of cytotoxic lymphocytes into solid tumours and insufficient cytotoxicity of myeloid cells.

Therapies 103

Therapies DNA Engineering Virus 103

DrugBank

JUNE 13, 2024

Researchers are developing pH-sensitive nanoparticles that will release their drug cargo only in the acidic tumor microenvironment, a feature of many solid tumors.   Gene Therapy: Reprogramming the Body's Cellular Code    Gene therapy is an exciting field that treats diseases at their genetic roots.

Therapies 98

Therapies Drugs DNA Treatment 98

Advarra

AUGUST 8, 2023

Gene therapy research is booming in the clinical setting. In this blog, we summarize the growth, risks, and regulatory requirements for gene therapy research. We also discuss how a centralized biosafety review process can benefit this type of research.

Therapies 52

Therapies Research Engineering Clinical Research 52

Drug Target Review

OCTOBER 26, 2023

Genome engineering and gene therapies that manipulate DNA sequences in cells have driven a biotechnological revolution over the past decade. 1 Adeno-associated virus (AAV) vectors are the leading platform for in vivo gene delivery for the treatment of various human diseases.

Engineering 105

Engineering Virus Therapies Immune Response 105

Vial

DECEMBER 11, 2023

Hepatitis C virus (HCV) infection is a global health problem with complications that place a significant economic burden on national health systems. Despite advances in clinical research, there remain significant challenges as only 20% of HCV cases are diagnosed, and of these, only 15% receive treatment.

Clinical Research 52

Clinical Research Therapies Research Vaccine 52

Alta Sciences

OCTOBER 7, 2024

Latest Findings in Gene Therapy Research blussier Mon, 10/07/2024 - 15:16 HTML A Promising Future for Gene Therapy Our experts have been diligently working on groundbreaking research, including a wide range of gene therapy studies, and have compiled a selection of scientific resources featuring the latest advancements.

Therapies 40

Therapies Research Laboratories Virus 40

Broad Institute

JUNE 10, 2024

The advance, from the lab of Broad core institute member David Liu , could one day help researchers develop a single gene therapy for diseases such as cystic fibrosis that are caused by one of hundreds or thousands of different mutations in a gene.

DNA 144

DNA Engineering Therapies Disease 144

SCIENMAG: Medicine & Health

JUNE 21, 2023

In an earlier clinical trial, in which the Bochum researchers were also involved, patients with chronic hepatitis E virus (HEV) infection were treated with the drug sofosbuvir, which was actually developed for the treatment of hepatitis C. […] Which viral variants circulate in the blood?

Therapies 72

Therapies Clinical Trials Virus Trials 72

PLOS: DNA Science

OCTOBER 12, 2023

In the final chapter of my 2012 book The Forever Fix: Gene Therapy and the Boy Who Saved It , I predicted that the technology would soon expand well beyond the rare disease world. Gene therapy clearly hasn’t had a major impact on health care, offering extremely expensive treatments for a few individuals with rare diseases.

Therapies 69

Therapies Immune Response DNA Virus 69

The Pharma Data

MARCH 23, 2022

To treat Ebola virus infections, researchers are taking a close look at a key piece of the virus: polymerase. Polymerase is a viral protein that directs how Ebola virus replicates its genome as it infects new hosts. Drugs that target polymerase could potentially treat Ebola virus infections and save lives.

Virus 52

Virus Small Molecule Protein Production Research 52

Broad Institute

SEPTEMBER 13, 2023

Developing diagnostics and treatments By Rose Circeo September 13, 2023 Breadcrumb Home Developing diagnostics and treatments At the Broad Institute, researchers are revealing deep biological insights about the causes of disease, which are leading to new ways to monitor, diagnose, and treat patients with greater precision.

Therapies 52

Therapies Engineering Virus Packaging 52

Drug Target Review

DECEMBER 5, 2024

Viral vectors have been crucial in transforming the gene therapy landscape due to their natural ability to infect cells. 1 In 2017, the US Food and Drug Administration (FDA) approved the first AAV-based gene replacement therapy (Luxturna), for Leber congenital amaurosis type 2.

Therapies 52

Therapies Virus DNA Compliance 52

DrugBank

MARCH 12, 2025

  The Expanding Role of mRNA in Cancer Therapy One of the most exciting applications of mRNA therapeutics lies in cancer treatment, where leveraging the immune system to target tumors offers a novel approach. These findings underscore the potential of mRNA to redefine cancer treatment by offering precise, patient-specific therapies.

Vaccine 97

Vaccine Treatment Clinical Trials Therapies 97

Drug Target Review

MAY 22, 2024

How does Circio’s circVec technology aim to enhance the potency and reduce the cost of current gold-standard gene therapy? There are three major reasons for why Circio has selected AATD as its lead gene therapy program. Firstly, there have been previous AAV gene therapy clinical studies in this indication that have shown promise.

Therapies 59

Therapies RNA Protein Expression DNA 59

DrugBank

JANUARY 29, 2025

Gene Therapy Gene therapy operates on the principle of modulating the DNA blueprint of cells to induce a therapeutic response. Gene therapy trials explore using lentiviral vectors to deliver a functional copy of the HBB gene to hematopoietic stem cells, the progenitors of all blood cells.

Therapies 52

Therapies DNA RNA Disease 52

The Pharma Data

APRIL 7, 2022

announced today that the companies have entered into a definitive agreement under which Pfizer will acquire ReViral, a privately held, clinical-stage biopharmaceutical company focused on discovering, developing, and commercializing novel antiviral therapeutics that target respiratory syncytial virus (RSV). NYSE: PFE) and ReViral Ltd.

Virus 52

Virus Vaccine Clinical Development Hospitals 52

Broad Institute

DECEMBER 13, 2023

By Leah Eisenstadt December 13, 2023 Two teams of Broad researchers produced detailed maps of the mouse nervous system. Related news Researchers map brain cell changes in Alzheimer’s disease Courtesy of the Chen and Macosko labs. Courtesy of the Chen and Macosko labs. Our results underscore the need to study them more deeply.”

DNA 140

DNA Virus Research Engineering 140

The Premier Consulting Blog

MARCH 7, 2024

The FDA’s January 2020 guidance, Chemistry, Manufacturing and Control (CMC) [1] Information for Human Gene Therapy Investigational New Drug Applications (INDs), outlines the analytical methods that define the quality, safety and efficacy of gene therapy therapeutics.

Production 52

Production Therapies DNA Virus 52

Codon

SEPTEMBER 29, 2024

It suggested that, in the future, certain classes of drugs, specifically genetic therapies, could be screened entirely virtually via models of life. The lab created a plan with eight research institutions across three continents. Models of life became the dominant research paradigm in nearly every life-sciences field.

Engineering 142

Engineering Virus DNA Therapies 142

Advarra

JANUARY 11, 2024

The applications of mRNA-based therapies in cancer research represent one of the next groundbreaking steps toward improved cancer treatments. While mRNA usage has played several roles in clinical research , oncology researchers in particular are eager to explore the possibilities of mRNA-based cancer vaccines.

Vaccine 52

Vaccine Therapies Clinical Trials Small Molecule 52

The Pharma Data

JANUARY 4, 2021

Collaboration with Biogen to develop gene therapy for an undisclosed target to treat inherited eye disease, plus option for additional target. 05, 2021 (GLOBE NEWSWIRE) — ViGeneron GmbH , a gene therapy company, today announced a global collaboration and licensing agreement with Biogen Inc. MUNICH, Germany, Jan.

Therapies 52

Therapies Virus Disease Licensing 52

Broad Institute

DECEMBER 6, 2023

A new, large-scale reference created by researchers at the Broad Institute of MIT and Harvard could help scientists and clinicians better understand the role of cytokines in health and disease. AC: This research could have far-reaching implications, accelerating our understanding of natural immune responses and human immune-related diseases.

Immune Response 138

Immune Response RNA Disease Medical Schools 138

Codon

MARCH 20, 2023

Signal Transduction and Targeted Therapy. Nucleic Acids Research. Molecular Therapy. Nucleic Acids Research. Nucleic Acids Research. Nucleic Acids Research. Gene Therapy. A new control switch could make RNA therapies easier to program. Szymczak P. Nature Communications. Krypotou E. Karlikow M.

Virus 52

Virus Engineering RNA Small Molecule 52

Advarra

MARCH 31, 2023

The use of engineered genetic materials in clinical trials is rapidly expanding, with potential applications for genetic vaccines, gene-modified cellular therapies, and gene therapies. Either way, occupational exposure to these gene delivery systems bears potential risks to the research staff.

Engineering 52

Engineering Clinical Research Research Virus 52

Chemical Biology and Drug Design

APRIL 18, 2023

Due to the aforementioned challenges, researchers are now focusing on discovering alternative novel safe, and economically reachable therapies for the treatment of trypanosomiasis. Abstract Twenty different infectious disorders induced by bacteria, viruses, and parasites are categorized as neglected tropical diseases (NTDs) by WHO.

Treatment 100

Treatment Disease Virus Therapies 100

Advarra

JUNE 27, 2023

Clinical trials are studies involving human volunteers to research ways to prevent, detect, and treat various medical conditions. A clinical trial investigating a drug or therapy is typically divided into four phases: Phase I, Phase II, Phase III, and Phase IV.

Clinical Research 52

Clinical Research Research Clinical Trials Trials 52

Alta Sciences

DECEMBER 12, 2024

Get Started on Your Gene Therapy Preclinical Studies Today! With a large population of NHPs that are pre-screened for adeno-associated viruses (AAVs), were ready to get started on your studies immediately. Speak with one of our experts today to get your project started right away!

Therapies 40

Therapies Virus Research 40

The Pharma Data

DECEMBER 29, 2020

Innovative, novel approach to treat and protect against SARS-CoV-2 using thermoresponsive virus-targeting nanoparticles as an inhalation medicine. Radboudumc, with nearly 9,000 employees and 3,000 students, is devoted to patient care, education and training, as well as performing excellent basic and clinical research. en/research.

Therapies 52

Therapies Virus Engineering International 52

Drug Target Review

DECEMBER 28, 2023

In most cancers, the tumour evolves by acquiring mutations that confer growth advantages or resistance to therapies. However, if the therapy targets only the subclonal (branch) mutations, this will result in the mere ‘pruning’ of specific branches rather than elimination of the whole cancer.

Bioinformatics 105

Bioinformatics Immune Response DNA Vaccine 105