Beyond factor replacement: gene therapy as a paradigm shift in haemophilia management (Record no. 22882)
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| 000 -LEADER | |
|---|---|
| fixed length control field | a |
| 003 - CONTROL NUMBER IDENTIFIER | |
| control field | OSt |
| 005 - DATE AND TIME OF LATEST TRANSACTION | |
| control field | 20250516114208.0 |
| 008 - FIXED-LENGTH DATA ELEMENTS--GENERAL INFORMATION | |
| fixed length control field | 250516b xxu||||| |||| 00| 0 eng d |
| 040 ## - CATALOGING SOURCE | |
| Original cataloging agency | AIKTC-KRRC |
| Transcribing agency | AIKTC-KRRC |
| 100 ## - MAIN ENTRY--PERSONAL NAME | |
| 9 (RLIN) | 26245 |
| Author | Khan, Insharah |
| 245 ## - TITLE STATEMENT | |
| Title | Beyond factor replacement: gene therapy as a paradigm shift in haemophilia management |
| 250 ## - EDITION STATEMENT | |
| Volume, Issue number | Vol.19(4), Oct-Dec |
| 260 ## - PUBLICATION, DISTRIBUTION, ETC. | |
| Place of publication, distribution, etc. | Jaipur |
| Name of publisher, distributor, etc. | Health Education Bureau |
| Year | 2024 |
| 300 ## - PHYSICAL DESCRIPTION | |
| Pagination | 11-21p. |
| 520 ## - SUMMARY, ETC. | |
| Summary, etc. | This review examines recent developments in Haemophilia treatment, with a focus on gene therapy approaches. Haemophilia A<br/>and B are genetic disorders caused by deficiencies in coagulation factors VIII and IX, respectively. While current treatments rely<br/>on regular infusions of clotting factors, they face limitations including frequent administration and the risk of inhibitor<br/>development. Gene therapy emerges as a promising alternative, offering the potential for long-term therapeutic gene expression.<br/>The review analyzes various gene therapy strategies, with particular attention to adeno-associated virus (AAV) vector-mediated<br/>approaches. Recent clinical trials have shown encouraging results in both the Haemophilia types, demonstrating sustained factor<br/>expression and reduced bleeding episodes in some patients.<br/>The paper discusses the complexities inherent in gene therapy, including immune responses, vector optimization, and safety<br/>considerations. It also explores genome editing technologies as potential future treatments. Additionally, the review touches on<br/>the economic implications of gene therapies, considering aspects of cost-effectiveness and health technology assessment.<br/>While gene therapy shows promise, challenges remain in ensuring long-term efficacy and safety. Ongoing research aims to<br/>address these issues, potentially transforming Haemophilia treatment from chronic management to a one-time curative approach.<br/>The review concludes by highlighting areas for further investigation and the potential impact of these therapies on Haemophilia<br/>care. |
| 650 #0 - SUBJECT ADDED ENTRY--TOPICAL TERM | |
| 9 (RLIN) | 7368 |
| Topical term or geographic name entry element | Hospital and Clinical Pharmacy |
| 700 ## - ADDED ENTRY--PERSONAL NAME | |
| 9 (RLIN) | 26248 |
| Co-Author | Mohd Farhan Shehzad |
| 773 0# - HOST ITEM ENTRY | |
| Place, publisher, and date of publication | Jaipur Health Education Bureau |
| Title | Journal of hospital pharmacy |
| International Standard Serial Number | 2348-7704 |
| 856 ## - ELECTRONIC LOCATION AND ACCESS | |
| URL | https://journalofhospitalpharmacy.in/johp/index.php?id=118&yr=13 |
| Link text | Click here |
| 942 ## - ADDED ENTRY ELEMENTS (KOHA) | |
| Source of classification or shelving scheme | Dewey Decimal Classification |
| Koha item type | Articles Abstract Database |
| Withdrawn status | Lost status | Source of classification or shelving scheme | Damaged status | Not for loan | Home library | Current library | Shelving location | Date acquired | Total Checkouts | Barcode | Date last seen | Price effective from | Koha item type |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Dewey Decimal Classification | School of Pharmacy | School of Pharmacy | Archieval Section | 16/05/2025 | 2025-0850 | 16/05/2025 | 16/05/2025 | Articles Abstract Database |