Gene Editing - A Cure for Transthyretin Amyloidosis?
Maurer, M.S.
New England Journal of Medicine 385(6): 558-559
2021
ISSN/ISBN: 0028-4793 PMID: 34347958 Accession: 071816633
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Sikora, J.L.; Logue, M.W.; Chan, G.G.; Spencer, B.H.; Prokaeva, T.B.; Baldwin, C.T.; Seldin, D.C.; Connors, L.H. 2015: Genetic variation of the transthyretin gene in wild-type transthyretin amyloidosis (ATTRwt) Human Genetics 134(1): 111-121
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Norgren, N.; Hellman, U.; Ericzon, B.G.ör.; Olsson, M.; Suhr, O.B. 2012: Allele specific expression of the transthyretin gene in swedish patients with hereditary transthyretin amyloidosis (ATTR V30M) is similar between the two alleles Plos one 7(11): E49981
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Briani, C.; Cavallaro, T.; Ferrari, S.; Taioli, F.; Calamelli, S.; Verga, L.; Adami, F.; Fabrizi, G.M. 2012: Sporadic transthyretin amyloidosis with a novel TTR gene mutation misdiagnosed as primary amyloidosis Journal of Neurology 259(10): 2226-2228
Suhr, O.B.; Svendsen, I.H.; Ohlsson, P.I.; Lendoire, J.; Trigo, P.; Tashima, K.; Ranløv, P.J.; Ando, Y. 1999: Impact of age and amyloidosis on thiol conjugation of transthyretin in hereditary transthyretin amyloidosis Amyloid: the International Journal of Experimental and Clinical Investigation: the Official Journal of the International Society of Amyloidosis 6(3): 187-191
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Bhuiyan, T.; Helmke, S.; Patel, A.R.; Ruberg, F.L.; Packman, J.; Cheung, K.; Grogan, D.; Maurer, M.S. 2011: Pressure-volume relationships in patients with transthyretin (ATTR) cardiac amyloidosis secondary to V122i mutations and wild-type transthyretin: Transthyretin Cardiac Amyloid Study (TRACS) CIRCULATION. Heart Failure 4(2): 121-128
Connors, L.H.; Prokaeva, T.; Lim, A.; Théberge, R.; Falk, R.H.; Doros, G.; Berg, A.; Costello, C.E.; O'Hara, C.; Seldin, D.C.; Skinner, M. 2009: Cardiac amyloidosis in African Americans: comparison of clinical and laboratory features of transthyretin V122I amyloidosis and immunoglobulin light chain amyloidosis American Heart Journal 158(4): 607-614