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AKV9, Protein Aggregation Inhibitor

Improving mitochondria and ER stability helps eliminate upper motor neuron degeneration that occurs due to mSOD1 toxicity and TDP-43 pathology
Genç B, Gautam M, Gözütok Ö, Dervishi I, Sanchez S, Goshu GM, Koçak N, Xie E, Silverman RB, Özdinler PH. Improving mitochondria and ER stability helps eliminate upper motor neuron degeneration that occurs due to mSOD1 toxicity and TDP-43 pathology. Clin Transl Med. 2021;11(2):e336.

Chiral cyclohexane 1,3-diones as inhibitors of mutant SOD1- dependent protein aggregation for the treatment of ALS
Zhang Y, Benmohamed R, Zhang W, Kim J, Edgerly CK, Zhu Y, Morimoto RI, Ferrante RJ, Kirsch DR, Silverman RB. Chiral cyclohexane 1,3-diones as inhibitors of mutant SOD1-dependent protein aggregation for the treatment of ALS. ACS Med Chem Lett. 2012; 3(7):584-587.

NU‑9 improves health of hSOD1G93A mouse upper motor neurons in vitro, especially in combination with riluzole or edaravone
Genç B., Gautam, M., Helmold, B. R., Koçak, N., Günay, A., Goshu, G. M., Silverman, R. B., & Hande Özdinler, P.  NU-9 improves health of hSOD1G93A mouse upper motor neurons in vitro, especially in combination with riluzole or edaravone. Sci Rep. 2022; 12(1), 5383.

Mitochondria, ER, and nuclear membrane defects reveal early mechanisms for upper motor neuron vulnerability with respect to TDP‑43 pathology
Gautam M, Jara JH, Kocak N, Rylaarsdam LE, Kim KD, Bigio EH, Hande Özdinler P. Mitochondria, ER, and nuclear membrane defects reveal early mechanisms for upper motor neuron vulnerability with respect to TDP-43 pathology. Acta Neuropathol. 2019;137(1):47-69.

Incorporating upper motor neuron health in ALS drug discovery.
Dervishi I, Özdinler PH. Incorporating upper motor neuron health in ALS drug discovery. Drug Discov Today. 2018 Mar;23(3):696-703.

Apical dendrite degeneration, a novel cellular pathology for Betz cells in ALS
Genç B, Jara JH, Lagrimas AK, Pytel P, Roos RP, Mesulam MM, Geula C, Bigio EH, Özdinler PH. Apical dendrite degeneration, a novel cellular pathology for Betz cells in ALS. Sci Rep. 2017;7:41765.

Pathophysiological and diagnostic implications of cortical dysfunction in ALS
Geevasinga N, Menon P, Özdinler PH, Kiernan MC, Vucic S. Pathophysiological and diagnostic implications of cortical dysfunction in ALS. Nat Rev Neurol. 2016;12(11):651-661.

Treatment of amyotrophic lateral sclerosis: lessons learned from many failures
Özdinler PH, Silverman RB. Treatment of amyotrophic lateral sclerosis: lessons learned from many failures. ACS Med Chem Lett. 2014;5(11):1179-81.

Moving forward in clinical trials for ALS: motor neurons lead the way please
Genç B, Özdinler PH. Moving forward in clinical trials for ALS: motor neurons lead the way please. Drug Discov Today. 2014 Apr;19(4):441-9.

eGFP expression under UCHL1 promoter genetically labels corticospinal neurons and a subpopulation of degeneration-resistant spinal motor neurons in an ALS mouse model
Yasvoina MV, Genç B, Jara JH, Sheets PL, Quinlan KA, Milosevic A, Shepherd GM, Heckman CJ, Özdinler PH. eGFP expression under UCHL1 promoter genetically labels corticospinal motor neurons and a subpopulation of degeneration-resistant spinal motor neurons in an ALS mouse model. J Neurosci. 2013 May 1;33(18):7890-904.

+ Expand all
AKV9, Protein Aggregation Inhibitor

Genç, B.; Gautam, M.; Gözütok, Ö.; Dervishi, I.; Sanchez, S.; Goshu, G. M.; Koçak, N.; Xie, E.; Silverman, R. B.; Özdinler, P. H. Improving mitochondria and ER stability helps eliminate upper motor neuron degeneration that occurs due to mSOD1 toxicity and TDP-43 pathology. Clin. Transl. Med., 2021, 11, e336.

Chiral cyclohexane 1,3-diones as inhibitors of mutant SOD1-dependent protein aggregation for the treatment of ALS

NU-9 improves health of hSOD1 G93A mouse upper motor neurons in vitro, especially in combination with riluzole or edaravone

Mitochondria, ER, and nuclear membrane defects reveal early mechanisms for upper motor neuron vulnerability with respect to TDP-43 pathology

Incorporating upper motor neuron health in ALS drug discovery

Apical dendrite degeneration, a novel cellular pathology for Betz cells in ALS

Pathophysiological and diagnostic implications of cortical dysfunction in ALS

Treatment of amyotrophic lateral sclerosis: lessons learned from many failures

Moving forward in clinical trials for ALS: motor neurons lead the way please

eGFP expression under UCHL1 promoter genetically labels corticospinal motor neurons and a subpopulation of degeneration-resistant spinal motor neurons in an ALS mouse model

Zhang,Y.; Benmohamed, R.; Zhang, W.; Kim, J.; Edgerly, C.K.; Zhu,Y.; Morimoto, R.I.; Ferrante ,R.J.; Kirsch, D.R.; Silverman, R.B. Chiral Cyclohexane 1,3-Diones as Inhibitors of Mutant SOD1- Dependent Protein Aggregation for the Treatment of ALS. ACS Med. Chem. Lett. 2012, 3, 584−587

Genç, B.; Mukesh, G.; Benjamin, H.R.; Koçak, N.; Günay, A.; Gashaw, G.M.; Silverman, R.B.; Özdinler, P.H. NU‑9 improves health of hSOD1G93A mouse upper motor neurons in vitro, especially in combination with riluzole or edaravone. Scientific Reports 2022, 12:5383

Gautam, M.; Javier, J.H.; Kocak, N.; Rylaarsdam, L.E.; Kim, K.D.,; Bigio, E.H.; Özdinler, P.H. Mitochondria, ER, and nuclear membrane defects reveal early mechanisms for upper motor neuron vulnerability with respect to TDP‑43 pathology. Acta Neuropathol. 2019 January ; 137(1): 47–69.

Dervishi, I.; Özdinler, P.H. Incorporating upper motor neuron health in ALS drug discovery. Drug Discov. Today. 2018 March ; 23(3): 696–703.

Genç, B.; Jara, J.H.; Largrimas, A.K.B.; Pytel, P.; Roos, R.P.; Mesulam, M.M.; Geula, C.; Bigio, E.H.;Ozdinler, P.H. Apical dendrite degeneration, a novel cellular pathology for Betz cells in ALS. Scientific Reports 2017, 7:41765.

Geevasinga, N.; Özdinler, P.H.; Kiernan, M.C.; Vucic,S. Pathophysiological and diagnostic implications of cortical dysfunction in ALS. Nat Rev Neurol. 2016 Nov;12(11):651-661.

Özdinler, P.H.; Silverman, R.B. 2014. Treatment of Amyotrophic Lateral Sclerosis: Lessons Learned from Many Failures ACS Med. Chem. Lett. 2014, 5, 1179−1181

Genç, B.; Özdinler, P.H. 2014. Moving forward in clinical trials for ALS: motor neurons lead the way please. Drug Discov. Today. 2014 April ; 19(4): 441–449.

Yasvoina, M.B.; Genç, B.; Jara, J.H.; Sheets, P.L.; Quinlan, K.A.; Milosevic, A.; Shepherd, G.M.G.; Heckman, C.J.; Özdinler, P.H. eGFP Expression under UCHL1 Promoter Genetically Labels Corticospinal Motor Neurons and a Subpopulation of Degeneration-Resistant Spinal Motor Neurons in an ALS Mouse Model. J. Neurosci., May 1, 2013. 33(18):7890 –7904.