Children's Hospital Los Angeles (CHLA)’s Post

I am very excited to share the recent pubblication of our most recent review titled "The New Frontiers of Gene Therapy and Gene Editing in Inflammatory Diseases". This review provides an overview of how genetic manipulation strategies are able to offer novel and transformative solutions for conditions that traditionally elude conventional treatments. We provide insight on how gene therapy and gene editing application can overcome the intricate interplay of genetic factors and immune responses which characterise inflammatory diseases. In addition, analysis of case studies and preclinical research show favourable results and substantial transformative impacts of gene-based therapies in patients and animal models of inflammatory diseases. #inflammation #genetherapy #geneediting #inflammatorydiseases

Please read this insightful article on the BLS Blog

Our review on Gene Therapy and Gene Editing in Inflammatory Disorder is out!

The field of gene therapy has seen significant progress in the past decade, with several treatments receiving official approvals and many more in clinical trials. However, a major limitation has been the inability to administer more than one dose of a virus carrying restorative genes, which limits the therapy's effectiveness. At the American Society of Gene and Cell Therapy annual meeting in May 2024, researchers presented various potential solutions to this problem. These include strategies to suppress immune responses, cloak the virus, or find alternatives to using the virus altogether. These innovations could lead to significant advancements in gene therapy, offering new possibilities for treating a wide range of diseases. #GeneTherapy #ASGCT #MedicalBreakthroughs #HealthInnovation #ClinicalTrials #Genetics #HealthTech #Immunology #Virology #HealthcareNews #Biotechnology

If you have a #hematologial disorder, this post is for you! #Casgevy, a gene editing therapy, holds promise for treating hematological disorders like sickle cell disease and beta-thalassemia. Using CRISPR-Cas9 technology, it targets and edits specific genes responsible for these conditions. In sickle cell disease, #Casgevy aims to correct the mutation in the hemoglobin gene, while in beta-thalassemia, it targets genes involved in the production of hemoglobin. For individuals with sickle cell disease or beta-thalassemia who rely on frequent transfusions and face complications, #Casgevy offers a curative approach by precisely editing the genetic instructions within their cells. The process can restore normal hemoglobin levels, reduce symptoms, and decrease the need for transfusions. Despite the progress, gene therapy is in its infancy, and there is much discussion about its costs, ethics, and accessibility for those in need. ---------------- I specialize in creating medical and scientific content in the areas of hematology🩸and genetics 🧬 Contact me if you require assistance with your upcoming project. https://lnkd.in/gXvNgMpZ #Casgevy #hematology #blood #raredisease #genetherapy #genetics #CRISPR #sicklecell

Researchers have developed a gene therapy approach using an advanced prime editing system to correct the F508del mutation, which causes cystic fibrosis. This technique allows precise editing of DNA to repair the mutation in patient-derived lung cells. The restored function of the edited cells matched current CF drug therapies, indicating the potential of gene therapy as a one-time treatment. However, the challenge remains in effectively delivering the gene-editing system to patient cells.

LucidQuest Strategic Insights (lqventures.com) >>> Gene&Cell Therapy >> Eye-drop version of Krystal Biotech's skin gene therapy helped restore vision of boy with rare disease: A reformulated version of Krystal Biotech’s gene therapy for “butterfly skin” helped restore the vision of a boy with a rare disease, which can cause scarring of the eye tissue in some patients. The 13-year-old boy — Antonio, according to the AP — has a rare genetic disease called dystrophic epidermolysis bullosa, or DEB, that disrupts the production of a type of collagen. It leads to skin that’s extremely fragile and that can blister or tear, like the wings of a butterfly. Around a quarter of these children also experience scarring of the eye, according to Krystal, which can potentially result in blindness. Krystal’s gene therapy for the disease is a gel called Vyjuvek, and was approved by the FDA last year. The herpesvirus-based therapy delivers functional copies of the collagen gene that’s mutated in patients with DEB. But unlike other approved gene therapies that can only be given once, Vyjuvek can be reapplied to the skin as appropriate. The experimental use of the therapy in Antonio’s eye came after his doctor, Alfonso Sabater of Bascom Palmer Eye Institute in Miami, reached out to Krystal Biotech to see if they could test Vyjuvek through a compassionate use program. According to a case report published in the New England Journal of Medicine, Antonio had undergone two surgeries for scar tissue on his eyes, but his sight continued to deteriorate in his right eye. In 2020, he enrolled in a Phase III study for Vyjuvek. In that study, Vyjuvek improved the healing of Antonio’s skin wounds, and his doctors hypothesized the treatment could be used to treat his eyes. Doctors performed surgery on Antonio’s right eye, and followed that up immediately with an eye-drop form of Vyjuvek. Afterwards, he received eye drops of the gene therapy three times a week for two weeks, and once a week from then on. Three months after surgery, the outermost layer of Antonio’s cornea was fully healed, though he continued to use the gene therapy eye drops monthly. Krystal’s next steps for the eye-drop version of Vyjuvek are up in the air. “We are in dialogue with the FDA to determine what would be required to support approval for an eye drop formulation,” the company told Endpoints News in an email. “Once we have agreement with the FDA, we will provide an update on our approval plans.” The biotech added that it could also expand the applications of its herpesvirus-based gene therapy platform to other eye diseases. #lucidquest #genetherapy #celltherapy

What is the complement system and how is it impacting the immune response to AAV gene therapy? We're thrilled to share this review article published earlier this year in Human Gene Therapy!

🔍 A Critical Hurdle in Gene Therapy! Despite tremendous advancements, gene therapy faces a significant challenge: the human immune system. Individuals like Donavon Decker, who suffers from a genetic muscle disorder, enter trials knowing they might never qualify for a second dose due to severe immune reactions. Even after 25 years, this issue remains unresolved. At a recent gene therapy conference, experts explored promising strategies to tackle this obstacle, including innovative immune suppression methods and non-viral delivery alternatives. Will these breakthroughs enable repeated treatments and expand access? The journey to find out is ongoing. #GeneTherapy #HealthcareInnovation #MedicalResearch