UC San Diego Health’s Post

Day 23 of #sicklecellawarenessmonth Is there a cure for #sicklecelldisease? Currently there is one approved curative treatment for #SickleCell Disease. Hematopoietic Stem Cell Transplantation. Unfortunately it is not available to all persons living with the disease because it's limited to only people who can find matching donors. A second option which is still under research is Gene Therapy, but since it's still being investigated, the only way to receive the treatment is by participating in a clinical trial. It's approval may introduce a curative treatment with greater accessibility. #sicklecellwarrior #sicklecellawareness

Now online!💻 MAGOH promotes #gastriccancer progression via hnRNPA1 expression inhibition-mediated RONΔ160/PI3K/AKT signaling pathway activation ----------------------------------------------------- Gastric cancer (GC) is associated with high mortality and heterogeneity and poses a great threat to humans. Gene therapies for the receptor tyrosine kinase RON and its spliceosomes are attracting increasing amounts of attention due to their unique characteristics. However, little is known about the mechanism involved in the formation of the RON mRNA alternative spliceosome RONΔ160. This study by Zhou et al revealed that MAGOH could promote the formation of RONΔ160 and activate the PI3K/AKT signaling pathway through the inhibition of hnRNPA1 expression. Full text👇 https://lnkd.in/d6rVjiDu #AlternativeSplicing #mRNA IFO - Istituto Nazionale Tumori Regina Elena - Istituto Dermatologico San Gallicano

#CARTcellTherapies in #MultipleMyeloma | Unleashing the Future 👏 | OPEN ACCESS Review from Paola Dama & Friends at Nature #Cancer Gene Therapy | The recent approval of the second FDA multiple myeloma CAR T-cell therapy has sparked immense promise in the field. Thus far, the results indicate its potential as a highly effective therapeutic solution. Moreover, ongoing preclinical and clinical trials are exploring the capabilities of CAR T-cells in targeting specific antigens on myeloma cells, offering hope for patients with relapsed/refractory MM (RRMM). These advances have shown the potential for CAR T cell-based medicines or combination therapies to elicit greater treatment responses and minimize side effects. In this context, it is crucial to delve into the history and functions of CAR T-cells while acknowledging their limitations. We can strategise and develop innovative approaches to overcome these barriers by understanding their challenges. This article* by Mohsen Sheykhhasan, Amirhossein Ahmadieh-Yazdi, Rosario Vicidomini, Naresh Poondla, PhD., MBA et al aims to provide insights into the application of CAR T-cells in treating MM, shedding light on their potential, limitations, and strategies employed to enhance their efficacy. *https://lnkd.in/efu3574X Celentyx Ltd Professor Nicholas Barnes PhD, FBPhS Omar Qureshi Catherine Brady FIGURE | Strategies to overcome challenges |

Intra-abdominal gene therapy utilizing AAV containing tumor-suppressor miRNA Reduced levels of microRNA (miR)-29b in peritoneal fluids are associated with peritoneal metastasis (PM) of gastric cancer. Researchers from Jichi Medical University in Shimotsuke, Japan, have demonstrated that intraperitoneal (IP) injection of AAV-miR-29b achieves robust transgene expression in peritoneal mesothelial cells (PMCs) and their exosomes, effectively suppressing mesothelial-to-mesenchymal transition (MMT) and peritoneal fibrosis, thereby inhibiting PM. Notably, AAV-miR-29b maintains its significant effects even when administered on day 3, and when combined with low-dose Paclitaxel (PTX) on day 7, it significantly reduces the number of PM nodules compared to PTX alone. This study suggests that intra-abdominal gene therapy using AAV containing tumor-suppressor miRNA, such as miR-29b, could serve as a promising strategy for both the prevention and treatment of PM in gastrointestinal cancers. https://lnkd.in/gVJc6S6F #genetherapy #gastriccancer #gastrointestinalcancer #aav #mirna #paclitaxel

🧬 𝐍𝐞𝐰 𝐅𝐃𝐀 𝐚𝐩𝐩𝐫𝐨𝐯𝐚𝐥 𝐟𝐨𝐫 𝐨𝐧𝐜𝐨𝐥𝐨𝐠𝐢𝐜𝐚𝐥 𝐚𝐩𝐩𝐥𝐢𝐜𝐚𝐭𝐢𝐨𝐧𝐬 𝐢𝐧 𝐭𝐡𝐞 𝐜𝐞𝐥𝐥 𝐚𝐧𝐝 𝐠𝐞𝐧𝐞 𝐭𝐡𝐞𝐫𝐚𝐩𝐲 𝐬𝐩𝐚𝐜𝐞 Last week marked a significant victory for 𝐈𝐨𝐯𝐚𝐧𝐜𝐞 𝐁𝐢𝐨𝐭𝐡𝐞𝐫𝐚𝐩𝐞𝐮𝐭𝐢𝐜𝐬, 𝐈𝐧𝐜., which secured FDA approval for 𝐀𝐌𝐓𝐀𝐆𝐕𝐈™ (𝐥𝐢𝐟𝐢𝐥𝐞𝐮𝐜𝐞𝐥) for the treatment of patients with skin cancer. This is the first individualised tumour-infiltrating lymphocyte (TIL) therapy to be introduced to the market, bringing the total number of CGT therapies in the US to 35. This approval, along with the recent EMA endorsement of 𝐂𝐚𝐬𝐠𝐞𝐯𝐲 by 𝐕𝐞𝐫𝐭𝐞𝐱 𝐏𝐡𝐚𝐫𝐦𝐚𝐜𝐞𝐮𝐭𝐢𝐜𝐚𝐥𝐬 for the European market, clearly indicates progress in the cell and gene therapy space.

The gene therapy nadofaragene firadenovec demonstrated durable clinical activity in patients with high-risk, BCG-unresponsive non-muscle–invasive bladder cancer with carcinoma in situ with or without papillary tumors (±Ta/T1). https://lnkd.in/e-ajfRPV

The Alliance for Regenerative Medicine Sector Snapshot highlights key cell and gene therapy sector data, provides an update on the 2024 clinical pipeline, and trends advancing cell therapy development. 🔵 My key takeaways: - The field is beginning to make progress outside #hematology indications as Iovance Biotherapeutics, Inc. received approval earlier this year for #Amtagvi to treat metastatic melanoma - Oncology has been the primary focus for cell therapies, but we are seeing an expansion into other disease areas, like autoimmune diseases - Within the clinical pipeline, cell therapies to treat solid tumors maintain a sizeable presence in early-stage trials, showing the potential for more therapies to reach the market in the coming years #celltherapy #genetherapy #cellandgenetherapy #advancedtherapies #autoimmunedisease #raredisease

Vikram M. Narayan, MD, highlights takeaways from 5-year follow-up data examining the gene therapy nadofaragene firadenovec and details promising emerging therapies for bladder cancer. Winship Cancer Institute of Emory University #oncology #bladdercancer https://lnkd.in/e_HWyYxV

As Rare Disease Day comes to a close in the US, let's reflect on the challenges faced by individuals with rare diseases like Osteogenesis Imperfecta (OI). This profoundly rare skeletal dysplasia affects an estimated 1 in 10,000 to 20,000 individuals worldwide, with 25,000-50,000 cases in the US alone. In a recent review, Alexandru Dinulescu and colleagues identified eight potential therapies beyond conventional treatments for OI. These alternative therapies offer promising prospects for patients, including bone marrow transplantation, anti-RANKL antibodies, sclerostin inhibitors, and gene therapy. These findings highlight the remarkable progress in OI research, yet underscore the ongoing need for exploration, especially in pediatric care. Collaboration among researchers, clinicians, and industry stakeholders is crucial in advancing toward a new era of OI treatment. Dedication to unraveling the complexities of OI and fostering innovative solutions is paramount to improving the lives of those affected. Let's continue to shed light on rare diseases like OI and work towards better treatments and outcomes. #OsteogenesisImperfecta #RareDisease #MedicalResearch #Innovation #Healthcare Read more about the findings here: https://lnkd.in/eKkxCcYf