Research graph
References from Autophagy activation via BAG3 gene therapy improves phenotype in a mouse model of LGMD1A. Local targets link to admitted publications; unresolved targets remain external evidence.
Myotilin is mutated in limb girdle muscular dystrophy 1A
10.1093/hmg/9.14.2141 · 2000 · External reference
Mutations in myotilin cause myofibrillar myopathy
10.1212/01.wnl.0000123576.74801.75 · 2004 · External reference
Clinical and myopathological evaluation of early- and late-onset subtypes of myofibrillar myopathy
10.1016/j.nmd.2011.05.002 · 2011 · External reference
Myotilin, a novel sarcomeric protein with two Ig-like domains, is encoded by a candidate gene for limb-girdle muscular dystrophy
10.1093/hmg/8.7.1329 · 1999 · External reference
Myotilin, the limb-girdle muscular dystrophy 1A (LGMD1A) protein, cross-links actin filaments and controls sarcomere assembly
10.1093/hmg/ddg020 · 2003 · External reference
The Z-disc proteins myotilin and FATZ-1 interact with each other and are connected to the sarcolemma via muscle-specific filamins
10.1242/jcs.02484 · 2005 · External reference
A class III PDZ binding motif in the myotilin and FATZ families binds enigma family proteins: a common link for Z-disc myopathies
10.1128/mcb.01454-08 · 2009 · External reference
Indications for a novel muscular dystrophy pathway. gamma-filamin, the muscle-specific filamin isoform, interacts with myotilin
10.1083/jcb.151.2.235 · 2000 · External reference
Myofibrillar myopathies
10.1016/b978-0-08-045031-5.00011-6 · 2011 · External reference
Myotilinopathy: refining the clinical and myopathological phenotype
10.1093/brain/awh576 · 2005 · External reference
Analysis of myotilin turnover provides mechanistic insight into the role of myotilinopathy-causing mutations
10.1042/bj20101672 · 2011 · External reference
BAG3 mediates chaperone-based aggresome-targeting and selective autophagy of misfolded proteins
10.1038/embor.2010.203 · 2011 · External reference
Breaking BAG: The Co-Chaperone BAG3 in Health and Disease
10.1016/j.tips.2016.04.007 · 2016 · External reference
Hsp70-Bag3 complex is a hub for proteotoxicity-induced signaling that controls protein aggregation
10.1073/pnas.1803130115 · 2018 · External reference
BAG3 directly interacts with mutated alphaB-crystallin to suppress its aggregation and toxicity
10.1371/journal.pone.0016828 · 2011 · External reference
Transgenic mice expressing the myotilin T57I mutation unite the pathology associated with LGMD1A and MFM
10.1093/hmg/ddl160 · 2006 · External reference
Construction and analysis of compact muscle-specific promoters for AAV vectors
10.1038/gt.2008.104 · 2008 · External reference
Protein Aggregates and Aggrephagy in Myopathies
10.3390/ijms24098456 · 2023 · External reference
Autophagy Defects in Skeletal Myopathies
10.1146/annurev-pathmechdis-012419-032618 · 2020 · External reference
Metformin rescues muscle function in BAG3 myofibrillar myopathy models
10.1080/15548627.2020.1833500 · 2021 · External reference
Dysregulated Autophagy and Sarcomere Dysfunction in Patients With Heart Failure With Co-Occurrence of P63A and P380S BAG3 Variants
10.1161/jaha.123.029938 · 2023 · External reference
Small heat shock protein B8: from cell functions to its involvement in diseases and potential therapeutic applications
10.4103/nrr.nrr-d-24-00517 · 2025 · External reference
The Role of the Heat Shock Protein B8 (HSPB8) in Motoneuron Diseases
10.3389/fnmol.2017.00176 · 2017 · External reference
The Role of the Multifunctional BAG3 Protein in Cellular Protein Quality Control and in Disease
10.3389/fnmol.2017.00177 · 2017 · External reference
Cooperation of a ubiquitin domain protein and an E3 ubiquitin ligase during chaperone/proteasome coupling
10.1016/s0960-9822(01)00487-0 · 2001 · External reference
BAG-1--a nucleotide exchange factor of Hsc70 with multiple cellular functions
10.1379/1466-1268(2003)008<0225:bnefoh>2.0.co;2 · 2003 · External reference
HspB8 chaperone activity toward poly(Q)-containing proteins depends on its association with Bag3, a stimulator of macroautophagy
10.1074/jbc.m706304200 · 2008 · External reference
Protein quality control during aging involves recruitment of the macroautophagy pathway by BAG3
10.1038/emboj.2009.29 · 2009 · External reference
Emerging roles of molecular chaperones and co-chaperones in selective autophagy: focus on BAG proteins
10.1007/s00109-011-0795-6 · 2011 · External reference
BAG3 induces the sequestration of proteasomal clients into cytoplasmic puncta: implications for a proteasome-to-autophagy switch
10.4161/auto.29409 · 2014 · External reference
RNAi-mediated Gene Silencing of Mutant Myotilin Improves Myopathy in LGMD1A Mice
10.1038/mtna.2014.13 · 2014 · External reference
Myofibrillar Myopathy - RETIRED CHAPTER, FOR HISTORICAL REFERENCE ONLY
1993 · External reference
Cardiac Phenotypes in Hereditary Muscle Disorders: JACC State-of-the-Art Review
10.1016/j.jacc.2018.08.2182 · 2018 · External reference
Novel Mutation in FLNC (Filamin C) Causes Familial Restrictive Cardiomyopathy
10.1161/circgenetics.117.001780 · 2017 · External reference
The novel αB-crystallin (CRYAB) mutation p.D109G causes restrictive cardiomyopathy
10.1002/humu.23248 · 2017 · External reference
A novel CRYAB mutation resulting in multisystemic disease
10.1016/j.nmd.2011.07.004 · 2012 · External reference
Dysregulated autophagy in restrictive cardiomyopathy due to Pro209Leu mutation in BAG3
10.1016/j.ymgme.2018.01.001 · 2018 · External reference
Mutation in BAG3 causes severe dominant childhood muscular dystrophy
10.1002/ana.21553 · 2009 · External reference
BAG3 myofibrillar myopathy presenting with cardiomyopathy
10.1016/j.nmd.2015.01.009 · 2015 · External reference
Unusual multisystemic involvement and a novel BAG3 mutation revealed by NGS screening in a large cohort of myofibrillar myopathies
10.1186/s13023-014-0121-9 · 2014 · External reference
Bag3 Regulates Mitochondrial Function and the Inflammasome Through Canonical and Noncanonical Pathways in the Heart
10.1016/j.jacbts.2022.12.013 · 2023 · External reference
BAG3-related myofibrillar myopathy: a further observation with cardiomyopathy at onset in pediatric age
2021 · External reference
Reengineering inducible cardiac-specific transgenesis with an attenuated myosin heavy chain promoter
10.1161/01.res.0000065442.64694.9f · 2003 · External reference
Transcriptional Targeting Approaches in Cardiac Gene Transfer Using AAV Vectors
10.3390/pathogens12111301 · 2023 · External reference
Design and testing of regulatory cassettes for optimal activity in skeletal and cardiac muscles
10.1007/978-1-61737-982-6_1 · 2011 · External reference
A Muscle Hybrid Promoter as a Novel Tool for Gene Therapy
10.1016/j.omtm.2019.09.001 · 2019 · External reference
AAV1.NT-3 gene therapy for charcot-marie-tooth neuropathy
10.1038/mt.2013.250 · 2014 · External reference
AAV1.NT-3 gene therapy for X-linked Charcot-Marie-Tooth neuropathy type 1
10.1038/s41434-021-00231-3 · 2021 · External reference
AAV1.NT-3 gene therapy in a CMT2D model: phenotypic improvements in GarsP278KY/+ mice
10.1093/braincomms/fcab252 · 2021 · External reference
AAV1.tMCK.NT-3 gene therapy improves phenotype in Sh3tc2-/- mouse model of Charcot-Marie-Tooth Type 4C
10.1093/braincomms/fcae394 · 2024 · External reference
AAV1.NT-3 gene therapy prevents age-related sarcopenia
2023 · External reference
AAV1.NT-3 gene therapy in the SOD1KO mouse model of accelerated sarcopenia
10.1002/jcsm.13303 · 2023 · External reference
AAV1.NT-3 gene therapy attenuates spontaneous autoimmune peripheral polyneuropathy
10.1038/gt.2015.67 · 2016 · External reference
AAV1.NT-3 gene therapy increases muscle fiber diameter through activation of mTOR pathway and metabolic remodeling in a CMT mouse model
10.1038/s41434-018-0009-8 · 2018 · External reference
AAV1.NT3 gene therapy mitigates the severity of autoimmune encephalomyelitis in the mouse model for multiple sclerosis
10.1038/s41434-025-00518-9 · 2025 · External reference
Systemic delivery of AAVrh74.tMCK.hCAPN3 rescues the phenotype in a mouse model for LGMD2A/R1
10.1016/j.omtm.2021.06.010 · 2021 · External reference
