Gene Editing — A Cure for Transthyretin Amyloidosis?
Imagine that you had several family members with transthyretin amyloidosis. In their sixth decade of life, numbness developed, initially in their hands and feet, that progressed proximally over a period of years. This numbness came with associated muscle weakness, and then symptoms of autonomic dysf...
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| Veröffentlicht in: | The New England journal of medicine 2021-08, Vol.385 (6), p.558-559 |
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| creator | Maurer, Mathew S |
| description | Imagine that you had several family members with transthyretin amyloidosis. In their sixth decade of life, numbness developed, initially in their hands and feet, that progressed proximally over a period of years. This numbness came with associated muscle weakness, and then symptoms of autonomic dysfunction developed, including profound dizziness with changes in posture and alternating constipation and diarrhea that hindered their ability to engage socially. With disease progression, a syndrome of heart failure with worsening fatigue, shortness of breath, and edema developed. They initially needed an assistive device to walk, and ultimately they could not ambulate at all, losing their . . . |
| doi_str_mv | 10.1056/NEJMe2110557 |
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In their sixth decade of life, numbness developed, initially in their hands and feet, that progressed proximally over a period of years. This numbness came with associated muscle weakness, and then symptoms of autonomic dysfunction developed, including profound dizziness with changes in posture and alternating constipation and diarrhea that hindered their ability to engage socially. With disease progression, a syndrome of heart failure with worsening fatigue, shortness of breath, and edema developed. They initially needed an assistive device to walk, and ultimately they could not ambulate at all, losing their . . .</description><identifier>ISSN: 0028-4793</identifier><identifier>EISSN: 1533-4406</identifier><identifier>DOI: 10.1056/NEJMe2110557</identifier><identifier>PMID: 34347958</identifier><language>eng</language><publisher>United States: Massachusetts Medical Society</publisher><subject>Amyloid Neuropathies, Familial - genetics ; Amyloid Neuropathies, Familial - therapy ; Amyloidosis ; Cardiology ; Cardiomyopathy ; Clinical outcomes ; Congestive heart failure ; Constipation ; Diarrhea ; Disease ; Drug therapy ; Edema ; Gene Editing ; Genetics ; Genetics General ; Genome editing ; Humans ; Myocarditis ; Neurology ; Neurosurgery ; Neurosurgery General ; Posture ; Proteins ; Transthyretin</subject><ispartof>The New England journal of medicine, 2021-08, Vol.385 (6), p.558-559</ispartof><rights>Copyright © 2021 Massachusetts Medical Society. 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They initially needed an assistive device to walk, and ultimately they could not ambulate at all, losing their . . .</description><subject>Amyloid Neuropathies, Familial - genetics</subject><subject>Amyloid Neuropathies, Familial - therapy</subject><subject>Amyloidosis</subject><subject>Cardiology</subject><subject>Cardiomyopathy</subject><subject>Clinical outcomes</subject><subject>Congestive heart failure</subject><subject>Constipation</subject><subject>Diarrhea</subject><subject>Disease</subject><subject>Drug therapy</subject><subject>Edema</subject><subject>Gene Editing</subject><subject>Genetics</subject><subject>Genetics General</subject><subject>Genome editing</subject><subject>Humans</subject><subject>Myocarditis</subject><subject>Neurology</subject><subject>Neurosurgery</subject><subject>Neurosurgery General</subject><subject>Posture</subject><subject>Proteins</subject><subject>Transthyretin</subject><issn>0028-4793</issn><issn>1533-4406</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2021</creationdate><recordtype>article</recordtype><sourceid>EIF</sourceid><sourceid>BEC</sourceid><sourceid>BENPR</sourceid><sourceid>GUQSH</sourceid><sourceid>M2O</sourceid><recordid>eNpt0LtOwzAUBmALgWgpbMwoEgwMBI5vSTyhqCoFVGApc-QkJ5Aql2InQzcegifkSTC0IITwYuv48y_rJ-SQwjkFGVzcT27vkFF3luEWGVLJuS8EBNtkCMAiX4SKD8ietQtwiwq1SwZccDeW0ZAEU2zQm-RlVzZP3vvrmxd7496gV7TGmxvd2O55ZdDdenG9qtoyb21pL_fJTqEriwebfUQerybz8bU_e5jejOOZn3ERdb7SAUtVqlWGmIcgsgIU8IwVkFIUXAGlgWBMpdwJSAtgOohUHsqc6sK94iNyus5dmvalR9sldWkzrCrdYNvbhEkZCRkICo4e_6GLtjeN-92XopRxJZ06W6vMtNYaLJKlKWttVgmF5LPP5Hefjh9tQvu0xvwHfxfowMka1LVNGlzU_-d8AE6keVY</recordid><startdate>20210805</startdate><enddate>20210805</enddate><creator>Maurer, Mathew S</creator><general>Massachusetts Medical Society</general><scope>CGR</scope><scope>CUY</scope><scope>CVF</scope><scope>ECM</scope><scope>EIF</scope><scope>NPM</scope><scope>AAYXX</scope><scope>CITATION</scope><scope>0TZ</scope><scope>7RV</scope><scope>7X7</scope><scope>7XB</scope><scope>8AO</scope><scope>8C1</scope><scope>8FE</scope><scope>8FH</scope><scope>8FI</scope><scope>ABUWG</scope><scope>AFKRA</scope><scope>AN0</scope><scope>AZQEC</scope><scope>BBNVY</scope><scope>BEC</scope><scope>BENPR</scope><scope>BHPHI</scope><scope>CCPQU</scope><scope>DWQXO</scope><scope>FYUFA</scope><scope>GHDGH</scope><scope>GNUQQ</scope><scope>GUQSH</scope><scope>HCIFZ</scope><scope>K0Y</scope><scope>LK8</scope><scope>M0R</scope><scope>M0T</scope><scope>M1P</scope><scope>M2M</scope><scope>M2O</scope><scope>M2P</scope><scope>M7P</scope><scope>MBDVC</scope><scope>NAPCQ</scope><scope>PQEST</scope><scope>PQQKQ</scope><scope>PQUKI</scope><scope>PRINS</scope><scope>PSYQQ</scope><scope>Q9U</scope><scope>7X8</scope></search><sort><creationdate>20210805</creationdate><title>Gene Editing — A Cure for Transthyretin Amyloidosis?</title><author>Maurer, Mathew S</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c348t-9a62b9ba9ceed704cf0903c2f0b1e43901164229b39ce0bf02a689d75d1afa9c3</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2021</creationdate><topic>Amyloid Neuropathies, Familial - genetics</topic><topic>Amyloid Neuropathies, Familial - therapy</topic><topic>Amyloidosis</topic><topic>Cardiology</topic><topic>Cardiomyopathy</topic><topic>Clinical outcomes</topic><topic>Congestive heart failure</topic><topic>Constipation</topic><topic>Diarrhea</topic><topic>Disease</topic><topic>Drug therapy</topic><topic>Edema</topic><topic>Gene Editing</topic><topic>Genetics</topic><topic>Genetics General</topic><topic>Genome editing</topic><topic>Humans</topic><topic>Myocarditis</topic><topic>Neurology</topic><topic>Neurosurgery</topic><topic>Neurosurgery General</topic><topic>Posture</topic><topic>Proteins</topic><topic>Transthyretin</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Maurer, Mathew S</creatorcontrib><collection>Medline</collection><collection>MEDLINE</collection><collection>MEDLINE (Ovid)</collection><collection>MEDLINE</collection><collection>MEDLINE</collection><collection>PubMed</collection><collection>CrossRef</collection><collection>Pharma and Biotech Premium PRO</collection><collection>Nursing & Allied Health Database</collection><collection>Health & Medical Collection</collection><collection>ProQuest Central (purchase pre-March 2016)</collection><collection>ProQuest Pharma Collection</collection><collection>Public Health Database</collection><collection>ProQuest SciTech Collection</collection><collection>ProQuest Natural Science Collection</collection><collection>Hospital Premium Collection</collection><collection>ProQuest Central (Alumni Edition)</collection><collection>ProQuest Central UK/Ireland</collection><collection>British Nursing Database</collection><collection>ProQuest Central Essentials</collection><collection>Biological Science Collection</collection><collection>eLibrary</collection><collection>ProQuest Central</collection><collection>Natural Science Collection</collection><collection>ProQuest One Community College</collection><collection>ProQuest Central Korea</collection><collection>Health Research Premium Collection</collection><collection>Health Research Premium Collection (Alumni)</collection><collection>ProQuest Central Student</collection><collection>Research Library Prep</collection><collection>SciTech Premium Collection</collection><collection>New England Journal of Medicine</collection><collection>ProQuest Biological Science Collection</collection><collection>Consumer Health Database</collection><collection>Healthcare Administration Database</collection><collection>Medical Database</collection><collection>ProQuest Psychology</collection><collection>Research Library</collection><collection>Science Database</collection><collection>Biological Science Database</collection><collection>Research Library (Corporate)</collection><collection>Nursing & Allied Health Premium</collection><collection>ProQuest One Academic Eastern Edition (DO NOT USE)</collection><collection>ProQuest One Academic</collection><collection>ProQuest One Academic UKI Edition</collection><collection>ProQuest Central China</collection><collection>ProQuest One Psychology</collection><collection>ProQuest Central Basic</collection><collection>MEDLINE - 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| source | MEDLINE; Elektronische Zeitschriftenbibliothek - Frei zugängliche E-Journals; New England Journal of Medicine |
| subjects | Amyloid Neuropathies, Familial - genetics Amyloid Neuropathies, Familial - therapy Amyloidosis Cardiology Cardiomyopathy Clinical outcomes Congestive heart failure Constipation Diarrhea Disease Drug therapy Edema Gene Editing Genetics Genetics General Genome editing Humans Myocarditis Neurology Neurosurgery Neurosurgery General Posture Proteins Transthyretin |
| title | Gene Editing — A Cure for Transthyretin Amyloidosis? |
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