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Therapy: Gene Therapy
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Total 31 results found since Jan 2013.

Cell-Based Gene Therapy for b-Thalassemia
Indian Pediatr. 2023 Apr 15;60(4):313-316.ABSTRACTThe United States Food and Drug Administration (FDA) approved betibeglogene autotemcel (beti-cel), the first cell-based gene therapy for adult and pediatric patients with b-thalassemia in August, 2022. This update details this and other novel therapies that have emerged in the treatment of b-thalassemia, apart from transfusion and iron chelation, with particular focus on newly approved gene therapy.PMID:37002845
Source: Indian Pediatrics - April 1, 2023 Category: Pediatrics Authors: Yajika Arya Puneet Kaur Sahi Source Type: research

The Future Of Vision And Eye Care
3D printed digital contact lenses, bionic eye implants, augmented reality: the future of vision and eye care is full of science fiction-sounding innovations. Here is where digital health will take ophthalmology in the future! More than 80 percent of perception comes through vision Researchers estimate that 80-85 percent of our perception, learning, cognition, and activities are mediated through vision. Compared to that, our hearing only processes 11 percent of information, while smell 3.5 percent, touch 1.5 percent and taste 1 percent. Don’t you think that’s possible? Renowned scholars, L.D. Ros...
Source: The Medical Futurist - May 10, 2022 Category: Information Technology Authors: berci.mesko Tags: Augmented Reality Cyborgization 3d printing AI diabetes digital digital health future guide Healthcare Innovation Personalized medicine technology vision eye care ophthalmology Source Type: blogs

Thalassemias: From gene to therapy
Mol Aspects Med. 2021 Oct 11:101028. doi: 10.1016/j.mam.2021.101028. Online ahead of print.ABSTRACTThalassemias (α, β, γ, δ, δβ, and εγδβ) are the most common genetic disorders worldwide and constitute a heterogeneous group of hereditary diseases characterized by the deficient synthesis of one or more hemoglobin (Hb) chain(s). This leads to the accumulation of unstable non-thalassemic Hb chains, which precipitate and cause intramedullary destruction of erythroid precursors and premature lysis of red blood cells (RBC) in the peripheral blood. Non-thalassemic Hbs display high oxygen affinity and no cooperativity. T...
Source: Molecular Medicine - October 15, 2021 Category: Molecular Biology Authors: Giovanna De Simone Alberto Quattrocchi Benedetta Mancini Alessandra di Masi Clara Nervi Paolo Ascenzi Source Type: research

Gene Therapies for Transfusion-dependent β-Thalassemia
Indian Pediatr. 2021 Mar 26:S097475591600302. Online ahead of print.ABSTRACTβ-Thalassemia is one of the most prevalent monogenic diseases usually caused by quantitative defects in the production of b-globin, a component of adult hemoglobin (a2b2), leading to severe anemia. Technological advances in genome sequencing, stem cell selection, viral vector development, transduction and gene-editing strategies now allow for efficient ex-vivo genetic manipulation of human hematopoietic stem cells that can lead to a meaningful clinical benefit in thalassemia patients. In this perspective, the status of the gene-therapy approaches ...
Source: Indian Pediatrics - March 27, 2021 Category: Pediatrics Authors: Sandeep Soni Source Type: research