Expressed protein ligation: a general method for protein engineering
A protein semisynthesis method—expressed protein ligation—is described that involves the
chemoselective addition of a peptide to a recombinant protein. This method was used to …
chemoselective addition of a peptide to a recombinant protein. This method was used to …
Gene therapy for metachromatic leukodystrophy
…, P Aubourg, RG Crystal, D Sondhi - Journal of …, 2016 - Wiley Online Library
Leukodystrophies (LDs) are rare, often devastating genetic disorders with neurologic symptoms.
There are currently no disease‐specific therapeutic approaches for these diseases. In …
There are currently no disease‐specific therapeutic approaches for these diseases. In …
Treatment of late infantile neuronal ceroid lipofuscinosis by CNS administration of a serotype 2 adeno-associated virus expressing CLN2 cDNA
S Worgall, D Sondhi, NR Hackett, B Kosofsky… - Human gene …, 2008 - liebertpub.com
Late infantile neuronal ceroid lipofuscinosis (LINCL) is an autosomal recessive, neurodegenerative
lysosomal storage disease affecting the CNS and is fatal by age 8 to 12 years. A total …
lysosomal storage disease affecting the CNS and is fatal by age 8 to 12 years. A total …
Enhanced survival of the LINCL mouse following CLN2 gene transfer using the rh. 10 rhesus macaque-derived adeno-associated virus vector
D Sondhi, NR Hackett, DA Peterson, J Stratton, M Baad… - Molecular therapy, 2007 - cell.com
… Scale bar in a indicative of scale in a–c, e–g, i–k, m–o and scale bar in d indicative of scale
in d, h, l, and p. (q) The remaining animals in each experimental group (n=3/group) were also …
in d, h, l, and p. (q) The remaining animals in each experimental group (n=3/group) were also …
Genetic modification of the lung directed toward treatment of human disease
Genetic modification therapy is a promising therapeutic strategy for many diseases of the
lung intractable to other treatments. Lung gene therapy has been the subject of numerous …
lung intractable to other treatments. Lung gene therapy has been the subject of numerous …
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 …
D Sondhi, L Johnson, K Purpura, S Monette… - … Gene Therapy, Part B …, 2012 - liebertpub.com
… Sondhi and colleagues demonstrate that … –D) Higher magnification of the regions indicated
by black boxes in (A): (B) site of administration; (C) region adjacent to site of administration; (D…
by black boxes in (A): (B) site of administration; (C) region adjacent to site of administration; (D…
Human iPSC models of neuronal ceroid lipofuscinosis capture distinct effects of TPP1 and CLN3 mutations on the endocytic pathway
…, KB Sims, JF Gusella, D Sondhi… - Human molecular …, 2014 - academic.oup.com
Neuronal ceroid lipofuscinosis (NCL) comprises ∼13 genetically distinct lysosomal disorders
primarily affecting the central nervous system. Here we report successful reprograming of …
primarily affecting the central nervous system. Here we report successful reprograming of …
Intracranial delivery of CLN2 reduces brain pathology in a mouse model of classical late infantile neuronal ceroid lipofuscinosis
…, J Bu, W Yang, Q Zhao, D Sondhi… - Journal of …, 2006 - Soc Neuroscience
… Purkinje cells were labeled with calbindin D monoclonal antibody (Sigma, St. Louis, MO) at
1:… Calbindin D- and GFAP-positive cells were detected with donkey anti-mouse secondary …
1:… Calbindin D- and GFAP-positive cells were detected with donkey anti-mouse secondary …
Disease characteristics and progression in patients with late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease: an observational cohort study
…, RG Crystal, A Kohlschütter, D Sondhi… - The Lancet Child & …, 2018 - thelancet.com
… Motor-language score data for DEM-CHILD comparison cohort 1 (A), WCMC comparison
cohort 1 (B), DEM-CHILD comparison cohort 2 (C), and WCMC comparison cohort 2 (D). Each …
cohort 1 (B), DEM-CHILD comparison cohort 2 (C), and WCMC comparison cohort 2 (D). Each …
Neurological deterioration in late infantile neuronal ceroid lipofuscinosis
…, B Kosofsky, MG Kaplitt, MM Souweidane, D Sondhi… - Neurology, 2007 - AAN Enterprises
Background: Late infantile neuronal ceroid lipofuscinosis (LINCL) is associated with
progressive degeneration of the brain and retina starting in early childhood. Methods: Thirty-two …
progressive degeneration of the brain and retina starting in early childhood. Methods: Thirty-two …
