University of Wisconsin-Madison researchers targeting a group of hereditary neurodegenerative diseases have found success using a gene therapy treatment in an animal model. The approach, which uses CRISPR-Cas9 genome editing technology, offers a unique and promising strategy that could one day treat rare but debilitating motor neuron diseases in humans.

A team of researchers report findings from a Phase I/II gene therapy clinical trial for patients with Leber congenital amaurosis, which found that the gene

A team of researchers report findings from a Phase I/II gene therapy clinical trial for patients with Leber congenital amaurosis, which found that the gene

The international technology group Körber is driving the integration of supply chain and data orchestration platforms into its Werum PAS-X Manufacturing Execution System (MES).

Location: Engineering Library- R857.P6P599 2016

WHEN you meet Peter Chalouhy it is hard to imagine the bubbly little boy may not make it to his 14th birthday. A stem cell gene therapy trial is his only hope but funds are needed to bring it here.

Two of the preeminent researchers of gene therapy hope to improve their patients' sight in an experimental operation (Stephen Voss/WPN)

Title: First Gene Therapy Approved in U.S.
Category: Health News
Created: 8/30/2017 12:00:00 AM
Last Editorial Review: 8/31/2017 12:00:00 AM

Title: Gene Therapy Makes Inroads Against a Form of Hemophilia
Category: Health News
Created: 7/22/2022 12:00:00 AM
Last Editorial Review: 7/22/2022 12:00:00 AM

Neurogene’s Rett syndrome gene therapy has preliminary data supporting safety and efficacy of the one-time treatment. But a late-breaking report of a serious complication in a patient who received the high dose sent shares of the biotech downward.

The post Neurogene Gene Therapy Shows Signs of Efficacy in Small Study, But an Adverse Event Spooks Investors appeared first on MedCity News.

With FDA approval, UCSF Benioff Children's Hospital Oakland pioneers gene therapy for beta thalassemia, a rare genetic blood disorder, potentially alleviating

Nobel Prize-winning inventors of CRISPR, awarded in 2020, have seen their gene-editing tool gain approval in the UK for the first-ever therapy targeting

Two girls with a rare genetic brain disorder called Rett Syndrome was successfully treated by a first-ever novel gene therapy- NGN-401, developed by US-based Neurogene.

CDD - CDKL5 deficiency disorder, a most common type of medlinkgenetic epilepsy/medlink in children, occurs due to the loss of genes producing the CDKL5 enzyme.

Patients born with genetic medlinkblindness/medlink experienced significant visual improvements after a single treatment with gene therapy developed

medlinkChildhood obesity/medlink, affecting nearly 20% of children and teens, has severe implications for their health, increasing the risk of medlinkarthritis/medlink,

The early peek at data from a Phase I/II study excited families, physicians, and investors 

Researchers are working to fix genetic conditions like Gaucher disease before birth by testing experimental therapies on fetal animals

Title: Study: Gene Therapy for HIV Safe, But Effectiveness Still Unclear
Category: Health News
Created: 5/2/2012 4:05:00 PM
Last Editorial Review: 5/3/2012 12:00:00 AM

Title: Gene Therapy May Help Fight Tough-to-Treat Blood Cancer
Category: Health News
Created: 5/1/2019 12:00:00 AM
Last Editorial Review: 5/2/2019 12:00:00 AM

Although studies of mixed chimerism following hematopoietic stem cell transplantation in patients with sickle cell disease (SCD) may provide insights into the engraftment needed to correct the disease and into immunological reconstitution, an extensive multilineage analysis is lacking. We analyzed chimerism simultaneously in peripheral erythroid and granulomonocytic precursors/progenitors, highly purified B and T lymphocytes, monocytes, granulocytes and red blood cells (RBC). Thirty-four patients with mixed chimerism and ≥12 months of follow-up were included. A selective advantage of donor RBC and their progenitors/precursors led to full chimerism in mature RBC (despite partial engraftment of other lineages), and resulted in the clinical control of the disease. Six patients with donor chimerism <50% had hemolysis (reticulocytosis) and higher HbS than their donor. Four of them had donor chimerism <30%, including a patient with AA donor (hemoglobin >10 g/dL) and three with AS donors (hemoglobin <10 g/dL). However, only one vaso-occlusive crisis occurred with 68.7% HbS. Except in the patients with the lowest chimerism, the donor engraftment was lower for T cells than for the other lineages. In a context of mixed chimerism after hematopoietic stem cell transplantation for SCD, myeloid (rather than T cell) engraftment was the key efficacy criterion. Results show that myeloid chimerism as low as 30% was sufficient to prevent a vaso-occlusive crisis in transplants from an AA donor but not constantly from an AS donor. However, the correction of hemolysis requires higher donor chimerism levels (i.e. ≥50%) in both AA and AS recipients. In the future, this group of patients may need a different therapeutic approach.

To date, more than 200 monogenic, often devastating, skin diseases have been described. Because of unmet medical needs, development of long-lasting and curative therapies has been consistently attempted, with the aim of correcting the underlying molecular defect. In this review, we will specifically address the few combined cell and gene therapy strategies that made it to the clinics. Based on these studies, what can be envisioned for the future is a patient-oriented strategy, built on the specific features of the individual in need. Most likely, a combination of different strategies, approaches, and advanced therapies will be required to reach the finish line at the end of the long and winding road hampering the achievement of definitive treatments for genodermatoses.

Researchers at Washington University School of Medicine in St. Louis found that gene therapy in mice helped build strength and significant muscle mass quickly, while reducing the severity of osteoarthritis. The gene therapy also prevented obesity, even when the mice were fed a high-fat diet.

Originally published in February 1967

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This blog post was written by Deanna Petersen, CBO of AVROBIO, as part of the From The Trenches feature of LifeSciVC. When AVROBIO went public in June 2018, I found myself on the steep end of an unexpected but interesting

The post Recipe For Managing Data Disclosure Successfully With Academic Partners: A Public Gene Therapy Company Perspective appeared first on LifeSciVC.

Guangping Gao, the head of the Horae Gene Therapy Center at the University of Massachusetts Medical School, will partner with Philadelphia-based Spark Therapeutics to figure out better ways to get disease-curing genes into cells. The collaboration, announced this morning, gives Spark (Nasdaq: ONCE) the option for an exclusive, world-wide license for any intellectual property to come out of it. No financial terms were disclosed. Earlier this year, Gao was featured in Newsweek magazine for seemingly…

BioMarin Pharmaceutical is boosting its early-stage pipeline by penning a deal with Swiss startup Dinaqor.

Scientists have discovered a new approach in which three different gene delivery vectors were injected intravenously and directly into the kidneys of mice.

Gene therapy to treat aldehyde dehydrogenase type 2 (ALDH2) deficiency helps prevent increased risk for esophageal cancer and osteoporosis linked to chronic alcohol exposure, revealed study.

Gene therapy could prevent or reverse cardiac dysfunction in Barth syndrome according to the new research at Boston Children's Hospital. The findings,

New study has found a recently developed system for switching on the activity of genes that could improve treatments for a broad range of neurological diseases.

Gene therapy could improve quality of life for many glaucoma patients. The research team tested a new approach that could provide additional treatment options and benefits.

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Researchers said the hope is that gene therapy will free patients from nearly monthly eye injections by offering a potential "one-and-done" treatment.

Kimberly Dunham-Snary discusses the long-term health considerations of gene therapy for mitochondrial diseases and David Grimm talks about the smell of death, Mercury crashing, and animal IQ. Hosted by Susanne Bard. [Image credit: Ben Gracewood CC BY-NC 2.0, via flickr]

This week we hear stories on how the sloshing of Earth’s core may spike major earthquakes, carbon monoxide’s role in keeping deep diving elephant seals oxygenated, and a festival celebrating heavily researched yet completely nonsensical theories with Online News Editor David Grimm. Sarah Crespi interviews staff writer Jocelyn Kaiser about the status of gene therapy, including a newly tested gene-delivering virus that may give scientists a new way to treat devastating spinal and brain diseases. Listen to previous podcasts.    [Image: Robert Schwemmer, CINMS, NOAA; Music: Jeffrey Cook]