Gene therapy for cross-correction of somatic organs and the CNS in mucopolysaccharidosis II in rodents and non-human primates

Authors: Chen, Nancy, Ehmann, David E., Crooker, Robert, Derakhchan, Katayoun, Fang, Xiaodong, Felice, Brian, Galbreath, Elizabeth J., Glaus, Charles, Gu, Hongbo, Huang, Yan, Li, Christine, Li, Xing, Liu, Nan, Palmieri, Kathleen, Simic, Damir, Sypek, Joseph, Thompson, Susan, Winkelmann, Christopher T., Choi, Vivian W.

Source: In Molecular Therapy - Methods & Clinical Development 8 June 2023 29:286-302

Protein Engineering on Human Recombinant Follistatin: Enhancing Pharmacokinetic Characteristics for Therapeutic Application

Authors: Shen, Chuan, Iskenderian, Andrea, Lundberg, Dianna, He, Tao, Palmieri, Kathleen, Crooker, Robert, Deng, Qingwei, Traylor, Matthew, Gu, Sheng, Rong, Haojing, Ehmann, David, Pescatore, Brian, Strack-Logue, Bettina, Romashko, Alla, Baviello, George, Gill, John, Zhang, Bohong, Meiyappan, Muthuraman, Pan, Clark, Norton, Angela W. ^*

Source: In The Journal of Pharmacology and Experimental Therapeutics August 2018 366(2):291-302

Myostatin and activin blockade by engineered follistatin results in hypertrophy and improves dystrophic pathology in mdx mouse more than myostatin blockade alone

Authors: Iskenderian, Andrea, Liu, Nan, Deng, Qingwei, Huang, Yan, Shen, Chuan, Palmieri, Kathleen, Crooker, Robert, Lundberg, Dianna, Kastrapeli, Niksa, Pescatore, Brian, Romashko, Alla, Dumas, John, Comeau, Robert, Norton, Angela, Pan, Jing, Rong, Haojing, Derakhchan, Katayoun, Ehmann, David

Source: Skeletal muscle. 8(1):1-16

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100184387537.0x000001&indx=1&recIds=ETOCvdc_100184387537.0x000001

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Whole Body and CNS Biodistribution of rhHNS in Cynomolgus Monkeys after Intrathecal Lumbar Administration: Treatment Implications for Patients with MPS IIIA.

Authors: Jou-Ku Chung, Luying Pan, Palmieri, Kathleen, Youssef, Amir S., McCauley, Thomas G.

Source: International Journal of Molecular Sciences; Dec2017, Vol. 18 Issue 12, p2594, 16p

Subject Terms: SANFILIPPO syndrome, GENETIC disorders in children, COGNITION disorders, CENTRAL nervous system, BLOOD-brain barrier, PHARMACOKINETICS, CEREBROSPINAL fluid, HEPARAN sulfate, THERAPEUTICS

Biodistribution of idursulfase in cynomolgus monkeys after intrathecal-lumbar administration

Authors: Chung, Jou-Ku, Brown, Eilish, Crooker, Robert, Palmieri, Kathleen J., McCauley, Thomas G.

Source: In Molecular Genetics and Metabolism February 2016 117(2):S35-S35

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