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.

Over £800 million to support improved healthcare delivery and breakthroughs in new treatments

Patients up and down the country are set to benefit from innovative new treatments and improved delivery of health and care services following significant funding to support ground-breaking experimental medicine research and advance the UK's response to patient safety challenges.

14 Oct 2022

Sudden cardiac death risk can be determined by genetic score

A recent study published in the Journal of the American College of Cardiology found that a genome-wide polygenic score for coronary artery disease (GPSCAD) can predict sudden and/or arrhythmic death (SAD) risk in coronary artery disease (CAD) patients without severe systolic dysfunction.

3 Oct 2022

PerkinElmer unveils industry-first cell analysis solution to streamline cell and gene therapy research and manufacturing

PerkinElmer, Inc., (NYSE: PKI), a global leader committed to innovating for a healthier world, today launched the Cellaca® PLX Image Cytometry System, a first-of-its-kind benchtop platform that enables researchers to assess multiple Critical Quality Attributes (CQAs) of cell samples in a single automated workflow, including cell identity, quality and quantity.

From Revvity 29 Sep 2022

Astrea Bioseparations introduces Nereus LentiHERO, a fit-for-purpose solution for lentiviral vector purification

Astrea Bioseparations (“Astrea Bio”), a leading provider of novel purification and separation tools supporting the development of next-generation therapeutics, has announced today the commercial launch of its Nereus LentiHERO™ lentiviral vector (LVV) purification technology.

29 Sep 2022

Researchers to use $1 million grant for improving blood cancer diagnosis and treatment

Adelaide researchers will use a $1 million grant to improve the diagnosis and treatment of blood cancers, which kill 16 Australians every day.

28 Sep 2022

Genome editing approaches enable precise gene correction and disease rescue in inherited retinal diseases

In a new paper, University of California, Irvine researchers explain how precision genome editing agents have enabled precise gene correction and disease rescue in inherited retinal diseases (IRDs).

23 Sep 2022

Scientists create new tool to identify genomic safe harbor sites in specific tissues

Scientists at St. Jude Children's Research Hospital have created a tool that can find safe places to introduce genes into human DNA.

22 Sep 2022

Monon Bioventures receives SBIR grant to develop glioblastoma therapeutic created at Purdue

Monon Bioventures LLC has received a one-year, $398,314 Phase I Small Business Innovation Research, or SBIR grant from the National Cancer Institute to demonstrate the feasibility of manufacturing a glioblastoma therapeutic created at the Purdue University College of Pharmacy.

22 Sep 2022

New imaging approach could help accelerate the development of new treatments for eye diseases

Researchers have developed a simple and fast way to perform optoretinography, an imaging technique that measures light-induced functional activity in the eye's retina, the network of neurons in the back of our eyes responsible for detecting light and initiating vision.

22 Sep 2022

New board members bring significant leadership experience in the biotech sector

Aspect Biosystems, a biotechnology company pioneering the development of bioprinted tissue therapeutics to transform how we treat disease, is pleased to announce the appointment of three new members to its Board of Directors: Dr. Nancy Krieger, Dr. Don Haut, and Dr. Devyn Smith.

From Aspect Biosystems 22 Sep 2022

New genetically-targeted MND therapy could be a turning point for patient care

Scientists believe a new genetically-targeted therapy to treat motor neurone disease (MND) could be a turning point for patient care, after the results of a Phase 3 clinical trial showed significant physical benefits for patients after 12 months.

21 Sep 2022

Study identifies a promising target for polycystic kidney disease treatment

Blocking the inhibition of PKD1 and PKD2 gene expression by deleting a binding site for microRNAs hindered the formation and growth of kidney cysts in autosomal dominant polycystic kidney disease (ADPKD) models, UT Southwestern researchers reported.

16 Sep 2022

Treating the cerebrospinal fluid to protect the brain from chemotherapy's toxic effects

Up to three quarters of patients receiving cancer chemotherapy suffer from "chemo brain" -; a long-term side effect that makes it harder to remember things, maintain attention, and learn new information. When it strikes children, whose brains are still developing, their schoolwork and self-esteem may suffer.

13 Sep 2022

New gene therapy approach could help prevent blindness in children with Leber congenital amaurosis

Researchers from the National Eye Institute have developed a gene therapy that rescues cilia defects in retinal cells affected by a type of Leber congenital amaurosis (LCA), a disease that causes blindness in early childhood.

8 Sep 2022

Combined stem cell and gene therapy shows promise to treat patients with ALS

Cedars-Sinai investigators have developed an investigational therapy using support cells and a protective protein that can be delivered past the blood-brain barrier.

5 Sep 2022

Alternative approaches to functional and phenotypic assays using multi-parameter flow cytometry

Synexa is an industry-leading biomarker and large molecule bio analytic research service provider. In this interview, NewsMedical speaks with Natalie Strickland, a principal scientist at Synexa, about alternative approaches to flow cytometry.

From Synexa Life Sciences BV 5 Sep 2022

Understanding how microglia change their states to adapt to different areas of the brain

In this interview, News Medical speaks to Jeffrey Stogsdill, Ph.D., about his latest research exploring how microglia change their state to adapt to different areas of the brain.

2 Sep 2022

New approach using CRISPR can engineer massive quantities of cells for therapeutic applications

A new variation of the CRISPR-Cas9 gene editing system makes it easier to re-engineer massive quantities of cells for therapeutic applications.

25 Aug 2022

Next generation mRNA vaccines exhibit excellent protective and therapeutic effects

Researchers around the world have been working for years on developing vaccines against different types of cancer, but without much success. Now, Tufts School of Engineering researchers think they have found one that does work. They have devised a method of targeting cancer in mice with a vaccine that is so strong and precise, it eliminates tumors and even prevents their recurrence.

16 Aug 2022

Higher doses of CAR-T therapy achieve better survival rates for young patients with hard-to-treat B-ALL

Young people who received doses of tisagenlecleucel, a chimeric antigen receptor T cell (CAR-T) therapy, at the higher end of the FDA-approved dosing range had significantly better survival rates at one year compared with those who received lower doses within this range, according to research published today in Blood Advances.

8 Aug 2022

Gene Therapy News and Research

Further Reading

Over £800 million to support improved healthcare delivery and breakthroughs in new treatments

Sudden cardiac death risk can be determined by genetic score

PerkinElmer unveils industry-first cell analysis solution to streamline cell and gene therapy research and manufacturing

Astrea Bioseparations introduces Nereus LentiHERO, a fit-for-purpose solution for lentiviral vector purification

Researchers to use $1 million grant for improving blood cancer diagnosis and treatment

Genome editing approaches enable precise gene correction and disease rescue in inherited retinal diseases

Scientists create new tool to identify genomic safe harbor sites in specific tissues

Monon Bioventures receives SBIR grant to develop glioblastoma therapeutic created at Purdue

New imaging approach could help accelerate the development of new treatments for eye diseases

New board members bring significant leadership experience in the biotech sector

New genetically-targeted MND therapy could be a turning point for patient care

Study identifies a promising target for polycystic kidney disease treatment

Treating the cerebrospinal fluid to protect the brain from chemotherapy's toxic effects

New gene therapy approach could help prevent blindness in children with Leber congenital amaurosis

Combined stem cell and gene therapy shows promise to treat patients with ALS

Alternative approaches to functional and phenotypic assays using multi-parameter flow cytometry

Understanding how microglia change their states to adapt to different areas of the brain

New approach using CRISPR can engineer massive quantities of cells for therapeutic applications

Next generation mRNA vaccines exhibit excellent protective and therapeutic effects

Higher doses of CAR-T therapy achieve better survival rates for young patients with hard-to-treat B-ALL

Sensing a Healthier Future

Achieving and maintaining ISO/IEC 27001 and BS 10012 certifications

Expanding research and clinical options for children with cancer

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