Dr Karda is leading gene therapy research for the development of novel gene therapies for Dravet syndrome, an incurable inherited childhood epilepsy. She recently obtained an LifeArc Philanthropic fund, in collaboration with Prof Simon Waddington, Prof Stephanie Schorge (UCL School of Pharmacy), Dr Gabriele Lignani (UCL Institute of Neurology) and Prof Helen Cross (UCL ICH Neurosciences Unit) to perform pre-clinical gene therapy studies for Dravet Syndrome. Dr Juan Antinao Diaz, Research Fellow is currently working on this project.

Dr Karda also has a GOSH Charity and Sparks National Funding to develop pre-clinical gene editing treatments for Dravet syndrome. In collaboration with Prof Schorge, Prof Waddington, Dr John Counsell (UCL GOS Institute of Child Health) & Dr Marc Moore (Royal Holloway Department of Biological Sciences). Dr Ellie Crompton, Research Fellow is currently working on this project.

NEURONAL ELONGATION FACTOR

In close collaboration with Dr Jo Ng (UCL Institute for Women’s Health), Prof Schorge & Prof Waddington, Dr Karda is also leading gene therapy research to develop new gene therapy strategies for a series of devastating childhood neurological diseases linked to mutations proteins associate with ribosomes.

METABOLIC DISEASES

In collaboration with Dr Counsel we are developing pre-clinical gene therapy treatments for rare metabolic disorders.

MITOCHONDRIAL DISEASES

In close collaboration with Prof Shamima Rahman (UCL GOS Institute of Child Health) & Prof Waddington we are working on projects to develop new gene therapy treatments for mitochondrial disorders. Dr Nandaki Keshavan Clinical Research Fellow is currently completing his PhD for gene therapy in a specific mitochondrial depletion syndrome.

NEUROMUSCULAR DISORDERS

In collaboration with Prof Pietro Fratta (UCL Queen Square Institute of Neurology) & Dr Counsell we are developing gene therapy treatments for neuromuscular disorders.

PUBLICATIONS

Ng J, Barral S, De La Fuente Barrigon C, Lignani G, Erdem FA, Wallings R, Privolizzi R, Rossignoli G, Alrashidi H, Heasman S, Meyer E, Ngoh A, Pope S, Karda R, Perocheau D, Baruteau J, Suff N, Antinao Diaz J, Schorge S, Vowles J, Marshall LR, Cowley SA, Sucic S, Freissmuth M, Counsell JR, Wade-Martins R, Heales SJR, Rahim AA, Bencze M, Waddington SN, Kurian MA. Gene therapy restores dopamine transporter expression and ameliorates pathology in iPSC and mouse models of infantile parkinsonism. Sci Transl Med. 2021 May 19;13(594):eaaw1564.

Counsell JR, De Brabandere G, Karda R, Moore M, Greco A, Bray A, Diaz JA, Perocheau DP, Mock U, Waddington SN. Re-structuring lentiviral vectors to express genomic RNA via cap-dependent translation. Molecular Therapy. Methods & Clinical Development. 2021 Mar 12;20:357.

Delhove JM, Karda R, FitzPatrick LM, Buckley SMK, Waddington SN, McKay TR (2020). "Non-invasive somatotransgenic bioimaging in living animals." F1000Research 9.

Diaz JA, Geard A, FitzPatrick LM, Delhove JM, Buckley SM, Waddington SN, McKay TR and Karda R, 2020. Continual Conscious Bioluminescent Imaging in Freely Moving Mice. In Bioluminescent Imaging (pp. 161-175). Methods Mol Biol. 2020

Suff N, Karda R, Diaz J.A, Ng J, Baruteau J, Perocheau D, Taylor PW, Alber, D, Buckley S.M, Bajaj-Elliott M and Waddington SN, 2019. Cervical gene delivery of the antimicrobial peptide, Human β-defensin (HBD)-3, in a mouse model of ascending infection-related preterm birth. Front. Immunol., 11 February 2020

Karda R, Rahim AA, Wong AM, Suff N, Diaz JA, Tijani M, Ng J, Baruteau J, Perocheau DP, Martin NP, Hughes M, Delhove JM, Counsell JR, Cooper JD, Henckaerts E, Mckay TR, Buckley SMK, Waddington SN. Generation of light-producing somatic-transgenic mice using adeno-associated virus vectors. Scientific Reports. 2020 Feb 7.

Karda R, Counsell JR, Karbowniczek K, Caproni LJ, Tite JP, Waddington SN. Production of lentiviral vectors using novel, enzymatically-produced, linear DNA. Nature Gene Therapy. 2019 Jan 14.

Suff N, Karda R, Diaz JA, Ng J, Baruteau J, Perocheau D, Tangney M, Taylor PW, Peebles D, Buckley SM, Waddington SN. Ascending Vaginal Infection Using Bioluminescent Bacteria Evokes Intrauterine Inflammation, Preterm Birth, and Neonatal Brain Injury in Pregnant Mice. The American journal of pathology. 2018 Jul 21.

Baruteau J, Perocheau DP, Hanley J, Lorvellec M, Rocha-Ferreira E, Karda R, Ng J, Suff N, Antinao Diaz J, Rahim A, Hughes MP, Banushi B, Prunty H, Hristova M, Ridout DA, Virasami A, Heales S, Howe SJ, Buckley SM, Mills PB, Gissen P, Waddington SN. Argininosuccinic aciduria fosters neuronal nitrosative stress reversed by Asl gene transfer. Nature Communication. 2018 August 29.

Counsell JR, Karda R, Antonio Diaz J, Carey L, Wiktorowicz T, Buckley SMK, Ameri S, Ng J, Baruteau J, Lindemann D, Rethwilm A, Rahim AA, Waddington SN, and Howe SJ. Foamy virus vectors transduce visceral organs and hippocampal structures following in vivo delivery to neonatal mice. Molecular Therapy, Nucleic Acids. 2018 August 3.

Delhove JMKM, Karda R, Hawkins KE, FitzPatrick LM, Waddington SN, McKay TR. Bioluminescence Monitoring of Promoter Activity In Vitro and In Vivo. Methods Mol Biol. 2017;1651:49-64

Karda R, Perocheau DP, Suff N, Ng, Delhove JM, Buckley SM, Richards S, Counsell JR, Hagberg HH, Johnson MR, McKay TR & Waddington SM. Continual conscious bioluminescent imaging in freely moving somatotransgenic mice. Scientific Reports. 7, 6374, 2017

Vink CA, Counsell JR, Perocheau DP, Karda R, Buckley SMK, Brugman MH, Galla M, Schambach A, McKay TM, Waddington SN and Howe SJ. Eliminating HIV-1 packaging sequences from lentiviral vector proviruses enhances safety and expedites gene transfer for gene therapy. Molecular Therapy, 2017.

Delhove JM, Buckley SM, Perocheau DP, Karda R, Arbuthnot P, Henderson NC, Waddington SN, McKay TR. Longitudinal in vivo bioimaging of hepatocyte transcription factor activity following cholestatic liver injury in mice. Scientific Reports. 2017;7.

Karda R, Buckley SMK, and Waddington SN. Gene therapy with AAV for cystic fibrosis. Am J Respir Crit Care Med. 2016;193(3):234-6.

Waddington SN, Privolizzi R, Karda R, O’Neill HC. A Broad Overview and Review of CRISPR-Cas Technology and Stem Cells. Current Stem Cell Reports. 2016;2(1):9-20.

Buckley SMK, Delhove JM, Perocheau DP, Karda R, Rahim AA, Howe SJ, Ward NJ, Birrell MA, Belvisi MG, Arbuthnot P, Johnson MR, Waddington SN, and McKay TR. (2015) In vivo bioimaging with tissue-specific transcription factor activated luciferase reporter. Scientific reports 5, 11842

Karda R, Buckley SMK, Mattar CM, Ng J, Massaro G, Hughes MP, Kurian MA, Baruteau J, Gissen P, Chan JKY, Bacchelli C, Waddington SN, and Rahim AA Perinatal systemic gene delivery using adeno-associated viral vectors. (2014) Frontiers in molecular neuroscience 7, 89

FURTHER INFORMATION

Dr Rajvinder Karda is also a board member of the British Gene and Cell Therapy Society. Also a member of the Advisory Committee for Spanish Dravet Foundation Charity.