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Subretinal Gene Therapy Improves Vision in Patients with Wet Age-Related Macular DegenerationTreatment with recombinant adeno-associated virus vector gene-therapy was shown to be safe and potentially effective in a small test group of patients with neovascular age-related macular degeneration.
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Taking Small Steps Toward an HIV CureResearchers have had little success in developing an HIV cure, but recent studies involving gene therapy, immune-based therapy, reactivation of the immune system, and "very early" treatment have produced promising results.
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How PCSK9 Moved from Gene to Therapy and What Comes NextIt wasn't long ago that researchers first identified a piece of DNA that regulates cholesterol. This gene has been recently developed into a cholesterol-lowering agent and it's time to address where we go from here.
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Updated CRYSTAL Data Support Effectiveness of Cetuximab (Erbitux) in Wild-Type KRAS Metastatic Colorectal CancerAt the 2010 Gastrointestinal Cancers Symposium, new data from the CRYSTAL trial identified BRAF gene mutations as a poor prognostic indicator in metastatic colorectal cancer (mCRC) but not predictive of response to therapy.
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Ronald Crystal, MD: The Possibilities of Gene Therapy in Neurodegenerative DiseaseThe chairman of the Department of Genetic Medicine at Weill Cornell Medicine detailed the ongoing research in developing gene therapy for patients with Alzheimer disease.
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FDA Approves Omidubicel to Reduce Infection Risk in Patients With Hematologic Malignancies Undergoing Stem Cell TransplantInfection rates in patients receiving omidubicel post-stem cell transplant were about 20% less than those in the control group.
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Zolgensma Gene Therapy Data Manipulation, GRF6019 Phase 2 Results in Mild to Moderate Alzheimer, Mind Moments PodcastNeurology News Network for the week ending August 10, 2019.
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Capsida Biotherapeutics' CAP-003 Displays Properties of Disease-Modification in Preclinical Parkinson Disease ResearchThe noninvasive gene therapy exceeded the 30% efficacy threshold for normalizing GCase activity across all doses.
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Adjuvant Olaparib Approved for BRCA+, High-Risk Early Breast CancerOlaparib is now the first early-line therapy targeting BRCA-mutations in breast cancer.
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Debating the True Benefit of Adjuvant TKIs in High-Risk RCCAdjuvant therapy with tyrosine kinase inhibitors for patients with high-risk renal cell carcinoma (RCC) who have undergone a nephrectomy may be supported by level IIa evidence from the National Comprehensive Cancer Network guidelines.
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CAR T-Cell Therapy Efficacious at 10 Years in Chronic Lymphocytic LeukemiaCarl June, MD, discussed the longest-term follow-up data on CAR T-cell therapy so far.
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Bispecific CD19/CD22 CAR T Cells Active in Pediatric Relapsed ALLTreatment with CD19/22 chimeric antigen receptor CAR T cells induced a promising response in patients with relapsed/refractory B-cell precursor acute lymphoblastic leukemia.
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More Research Needed to Understand COVID-19 Vaccine Effects in CAR T RecipientsFurther understanding of how COVID-19 vaccinations affect CAR T-cell therapy recipients is needed.
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BCMA/CD19 CAR T Shows Efficacy in Newly Diagnosed Multiple MyelomaUpdated data were presented at the ASH 2022 annual meeting.
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Februrary 2008 Clinical Trial ReportsThe Clinical Trials reported in this issue include: PHASE III: 1) What Factors Predict Prognosis in Patients With Malignant Pleural Mesothelioma? 2) Treatment for Patients With High-Risk Postoperative Breast Cancer PHASE II: 1) Epratuzumab Plus Rituximab Equals Non-Hodgkin's Lymphoma Response 2) Topotecan Added to Twice-Daily Chemoradiation to Treat Limited- Stage, Small-Cell Lung Cancer 3) First-Line Therapy for Patients With Advanced or Metastatic Non–Small-Cell Lung Cancer 4) Radiotherapy for Nasal Squamous Cell Carcinoma Therapy PHASE I/II: 1) A New Combination Approach for Advanced-Stage Germ-Cell Tumors
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Targeting Non-Mutant EGFR: Genetic and Clinical Biomarkers May Help Validate Use of InhibitorsA recent study suggests patients may be selected for therapy based on the number of EGFR gene copies, and evaluated for clinical benefit based on the severity of the rash that often develops.
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Gene Signatures Predict for Radiation Response in Breast CancerRecent studies have demonstrated that somatic mutations of ATM denote greater response to radiation therapy and that gene patterns may help predict the timing of recurrence following radiation.
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Using Polygenic Risk Score to Better Outcomes in Primary Open-Angle GlaucomaCurrent studies are using a polygenic risk score to evaluate the risk and clinical outcomes in primary open-angle glaucoma (POAG).
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Topical Gene Therapy B-VEC Improves Vision in Compassionate Use ProgramKrystal Biotech's B-VEC was well-tolerated and the treated patient experienced significant improvement in visual acuity.
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The State of Retina TherapeuticsAn international panel of experts discussed needs and challenges in the field.
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Gene therapy for wet AMD shows promising long-term resultsThree-year follow-up from the phase I/IIa trial of rAAV.sFlt-1 subretinal injection is encouraging for gene therapy for exudative age-related macular degeneration.
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Gene therapy for choroideremia may be on horizonGene therapy to treat choroideremia is one step closer to reality thanks to a new study.
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Researchers Highlight Potential of Gene Editing in Inherited Retinal DiseasesStudies have demonstrated positive data that suggest in vivo gene editing will be the future treatment paradigm for retinal diseases.
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Inherited leber's congenital amaurosis gene therapy may be effectiveA novel gene therapy approach for the treatment of a type of inherited retinal disease appears to be safe and effective in initial trials. Results offer hope for the treatment of a gamut of eye diseases that once were thought to be untreatable, according to one researcher.
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Biologic agents treat ocular inflammatory diseaseEditor's Note: As defined by the FDA, the term "biologics" refers to a wide range of products that includes vaccines, blood and blood components, allergenics, somatic cells, gene therapy, tissues, and recombinant therapeutic proteins, such as monoclonal antibodies and antibody fragments.
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Biologic agents treat ocular inflammatory diseaseEditor's Note: As defined by the FDA, the term "biologics" refers to a wide range of products that includes vaccines, blood and blood components, allergenics, somatic cells, gene therapy, tissues, and recombinant therapeutic proteins, such as monoclonal antibodies and antibody fragments.
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Biologic agents treat ocular inflammatory diseaseEditor's Note: As defined by the FDA, the term "biologics" refers to a wide range of products that includes vaccines, blood and blood components, allergenics, somatic cells, gene therapy, tissues, and recombinant therapeutic proteins, such as monoclonal antibodies and antibody fragments.
Latest:
Biologic agents treat ocular inflammatory diseaseEditor's Note: As defined by the FDA, the term "biologics" refers to a wide range of products that includes vaccines, blood and blood components, allergenics, somatic cells, gene therapy, tissues, and recombinant therapeutic proteins, such as monoclonal antibodies and antibody fragments.
