Expressed protein ligation: A general method for protein engineering

Por Muir, Tom W., Sondhi, Dolan, Cole, Philip A.
Publicado em 1998

When Ethics Constrains Clinical Research: Trial Design of Control Arms in “Greater Than Minimal Risk” Pediatric Trials

Por de Melo-Martín, Inmaculada, Sondhi, Dolan, Crystal, Ronald G.
Publicado em 2011

Novel Therapies, High-Risk Pediatric Research, and the Prospect of Benefit: Learning from the Ethical Disagreements

Por de Melo-Martín, Inmaculada, Sondhi, Dolan, Crystal, Ronald G
Publicado em 2012

Gene Therapy for Metachromatic Leukodystrophy

Por Rosenberg, Jonathan B., Kaminsky, Stephen M., Aubourg, Patrick, Crystal, Ronald G., Sondhi, Dolan
Publicado no J Neurosci Res (2016)

Survival Advantage of Neonatal CNS Gene Transfer for Late Infantile Neuronal Ceroid Lipofuscinosis

Por Sondhi, Dolan, Peterson, Daniel A., Edelstein, Andrew M., del Fierro, Katrina, Hackett, Neil R., Crystal, Ronald G.
Publicado em 2008

Intrapleural Gene Therapy for Alpha-1 Antitrypsin Deficiency-Related Lung Disease

Por Stiles, Katie M., Sondhi, Dolan, Kaminsky, Stephen M., De, Bishnu P., Rosenberg, Jonathan B., Crystal, Ronald G.
Publicado no Chronic Obstr Pulm Dis (2018)

Gene Therapy for C1 Esterase Inhibitor Deficiency in a Murine Model of Hereditary Angioedema

Por Qiu, Ting, Chiuchiolo, Maria J., Whaley, Adele S., Russo, Anthony R., Sondhi, Dolan, Kaminsky, Stephen M., Crystal, Ronald G., Pagovich, Odelya E.
Publicado no Allergy (2019)

Intra-Arterial Delivery of AAV Vectors to the Mouse Brain After Mannitol Mediated Blood Brain Barrier Disruption

Por Foley, Conor P., Rubin, David G., Santillan, Alejandro, Sondhi, Dolan, Dyke, Jonathan P., Crystal, Ronald G., Gobin, Y. Pierre, Ballon, Douglas J.
Publicado no J Control Release (2014)

Untargeted Metabolite Profiling of Cerebrospinal Fluid Uncovers Biomarkers for Severity of Late Infantile Neuronal Ceroid Lipofuscinosis (CLN2, Batten Disease)

Por Sindelar, Miriam, Dyke, Jonathan P., Deeb, Ruba S., Sondhi, Dolan, Kaminsky, Stephen M., Kosofsky, Barry E., Ballon, Douglas J., Crystal, Ronald G., Gross, Steven S.
Publicado no Sci Rep (2018)

Correction of Brain Oligodendrocytes by AAVrh.10 Intracerebral Gene Therapy in Metachromatic Leukodystrophy Mice

Por Piguet, Françoise, Sondhi, Dolan, Piraud, Monique, Fouquet, Françoise, Hackett, Neil R., Ahouansou, Ornella, Vanier, Marie-Thérèse, Bieche, Ivan, Aubourg, Patrick, Crystal, Ronald G., Cartier, Nathalie, Sevin, Caroline
Publicado em 2012

Comparative Efficacy and Safety of Multiple Routes of Direct CNS Administration of Adeno-Associated Virus Gene Transfer Vector Serotype rh.10 Expressing the Human Arylsulfatase A c...

Por Rosenberg, Jonathan B., Sondhi, Dolan, Rubin, David G., Monette, Sébastien, Chen, Alvin, Cram, Sara, De, Bishnu P., Kaminsky, Stephen M., Sevin, Caroline, Aubourg, Patrick, Crystal, Ronald G.
Publicado no Hum Gene Ther Clin Dev (2014)

Symmetric Age-association of Retinal Degeneration in Patients with CLN2-Associated Batten Disease

Por Kovacs, Kyle D, Patel, Samir, Orlin, Anton, Kim, Keunpyo, Van Everen, Sherri, Conner, Therese, Sondhi, Dolan, Kaminsky, Stephen M, D’Amico, Donald J, Crystal, Ronald G, Kiss, Szilárd
Publicado no Ophthalmol Retina (2020)

Partial Correction of the CNS Lysosomal Storage Defect in a Mouse Model of Juvenile Neuronal Ceroid Lipofuscinosis by Neonatal CNS Administration of an Adeno-Associated Virus Serot...

Por Sondhi, Dolan, Scott, Emma C., Chen, Alvin, Hackett, Neil R., Wong, Andrew M.S., Kubiak, Agnieszka, Nelvagal, Hemanth R., Pearse, Yewande, Cotman, Susan L., Cooper, Jonathan D., Crystal, Ronald G.
Publicado em 2014

Anti-Epidermal Growth Factor Receptor Gene Therapy for Glioblastoma

Por Hicks, Martin J., Chiuchiolo, Maria J., Ballon, Douglas, Dyke, Jonathan P., Aronowitz, Eric, Funato, Kosuke, Tabar, Viviane, Havlicek, David, Fan, Fan, Sondhi, Dolan, Kaminsky, Stephen M., Crystal, Ronald G.
Publicado no PLoS One (2016)

Vectored Intracerebral Immunization with the Anti-Tau Monoclonal Antibody PHF1 Markedly Reduces Tau Pathology in Mutant Tau Transgenic Mice

Por Liu, Wencheng, Zhao, Lingzhi, Blackman, Brittany, Parmar, Mayur, Wong, Man Ying, Woo, Thomas, Yu, Fangmin, Chiuchiolo, Maria J., Sondhi, Dolan, Kaminsky, Stephen M., Crystal, Ronald G., Paul, Steven M.
Publicado no J Neurosci (2016)

Disrupted Adenovirus-Based Vaccines Against Small Addictive Molecules Circumvent Anti-Adenovirus Immunity

Por De, Bishnu P., Pagovich, Odelya E., Hicks, Martin J., Rosenberg, Jonathan B., Moreno, Amira Y., Janda, Kim D., Koob, George F., Worgall, Stefan, Kaminsky, Stephen M., Sondhi, Dolan, Crystal, Ronald G.
Publicado em 2013

Long-Term Expression and Safety of Administration of AAVrh.10hCLN2 to the Brain of Rats and Nonhuman Primates for the Treatment of Late Infantile Neuronal Ceroid Lipofuscinosis

Por Sondhi, Dolan, Johnson, Linda, Purpura, Keith, Monette, Sebastien, Souweidane, Mark M., Kaplitt, Michael G., Kosofsky, Barry, Yohay, Kaleb, Ballon, Douglas, Dyke, Jonathan, Kaminksy, Stephen M., Hackett, Neil R., Crystal, Ronald G.
Publicado em 2012

Gene therapy for late infantile neuronal ceroid lipofuscinosis: neurosurgical considerations

Por Souweidane, Mark M., Fraser, Justin F., Arkin, Lisa M., Sondhi, Dolan, Hackett, Neil R., KaMinSky, Stephen M., Heier, Linda, Kosofsky, Barry E., Worgall, Stefan, Crystal, Ronald g., Kaplitt, Michael g.
Publicado em 2010

Disease characteristics and progression in patients with late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease: an observational cohort study

Por Nickel, Miriam, Simonati, Alessandro, Jacoby, David, Lezius, Susanne, Kilian, Dirk, Van de Graaf, Benjamin, Pagovich, Odelya E, Kosofsky, Barry, Yohay, Kaleb, Downs, Matthew, Slasor, Peter, Aiayi, Temitayo, Crystal, Ronald G, Kohlschütter, Alfried, Sondhi, Dolan, Schulz, Angela
Publicado no Lancet Child Adolesc Health (2018)

Spectrum of Ocular Manifestations in CLN2-Associated Batten (Jansky-Bielschowsky) Disease Correlate with Advancing Age and Deteriorating Neurological Function

Por Orlin, Anton, Sondhi, Dolan, Witmer, Matthew T., Wessel, Matthew M., Mezey, Jason G., Kaminsky, Stephen M., Hackett, Neil R., Yohay, Kaleb, Kosofsky, Barry, Souweidane, Mark M., Kaplitt, Michael G., D’Amico, Donald J., Crystal, Ronald G., Kiss, Szilárd
Publicado em 2013