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1 ic plague is a devastating and transmissible disease for which a Food and Drug Administration-approve
2 sis (ALS) is a devastating neurodegenerative disease for which a greater understanding of early disea
3 se in the context of dyslipidemia, a complex disease for which a large population of patients has bee
4 e used in the study of any neurodegenerative disease for which a mouse model exists, promising to rev
5                             It was the first disease for which a vaccine was developed and remains th
6 f COVID-19, can be readily extended to other diseases for which a dearth of chemical-chemical combina
7              We discuss here the examples of diseases for which a link with R loops has been describe
8 r metabolome profiling is patients with rare disease for which abnormal metabolic signatures may yiel
9 tis (AD) is a complex and heterogeneous skin disease for which achieving complete clinical clearance
10 ribute to the development and progression of diseases for which acute or chronic inflammation has bee
11 coholic hepatitis (AH) is a life-threatening disease for which adequate oral nutritional support is r
12 1 diabetes (T1D) is a devastating autoimmune disease for which advanced mass spectrometry (MS) method
13 cular mechanisms of aging and the origins of diseases for which age is a risk factor.
14 entifying therapeutics for neurodegenerative diseases for which aging is the biggest risk factor.
15                           Except for Gaucher disease, for which an enzyme with exposed mannosyl resid
16 s occur rapidly in kidney and cardiovascular disease, for which an in-line monitor could be beneficia
17 eficiency virus (HIV) is the only infectious disease for which anonymous testing is publicly funded,
18 nvasive mold infections are life-threatening diseases for which appropriate antifungal therapy is cru
19 of new therapeutic strategies for glomerular diseases for which available therapies are not fully eff
20 s with pulmonary tuberculosis, a devastating disease for which B-lactam drugs historically have been
21 cause-specific mortality from cardiovascular diseases for which background risk varies because of oth
22 adenocarcinoma (PDAC) is a highly metastatic disease for which better therapies are urgently needed.
23 hemia and pulmonary hypertension are 2 human diseases for which better therapies are needed.
24 antle cell lymphoma is a generally incurable disease for which blood or marrow transplantation is fre
25 h are far more numerous than the hematologic diseases for which CAR-Ts have been granted marketing au
26                  In contrast to Huntington's disease (for which ChAc has occasionally been mistaken),
27 ssociation with a second systemic autoimmune disease for which classification criteria are fulfilled.
28  of reassurance to many patients with HCM, a disease for which clinical course is often unfavorable a
29 e procedure, as first-step management in PAC disease, for which combined cataract and glaucoma surger
30 particularly appealing for neurodegenerative diseases, for which conventional pharmacologic approache
31 ome) is increasingly used for studying human disease, for which copy-number variants (CNVs) are a cri
32 cation of countermeasures for mpox and other diseases for which countermeasures are in short supply.
33 , particularly in extensively drug-resistant disease for which current antibiotic regimens are largel
34 on pancreatic ductal adenocarcinoma (PDA), a disease for which current therapeutic options are limite
35 ematosus (SLE) is an inflammatory autoimmune disease for which current therapy is suboptimal.
36 hic pulmonary fibrosis (IPF) is a fatal lung disease for which current treatment options only slow cl
37 hic pulmonary fibrosis (IPF) is a fatal lung disease for which current treatment options only slow cl
38 ng squamous cell carcinoma (SCC) is a deadly disease for which current treatments are inadequate.
39                 Leishmaniasis is a neglected disease, for which current treatment presents numerous i
40 arget of brain disorders such as Alzheimer's disease, for which currently no treatment is available.
41 icits, possibly reflecting neurodegenerative diseases for which currently no robust biomarkers are av
42 ngitis are common complications of gallstone disease for which data on current epidemiological trends
43 sclerosis (MS) is a heterogeneous autoimmune disease for which diagnosis continues to rely on subject
44 s, including regeneration, reprogramming and disease, for which direction is unknown.
45 antile onset represent an important class of disease for which discovery of the underlying genetic ar
46 ess, ALS is one of the few neurodegenerative diseases for which disease-modifying therapies are appro
47 ma gene therapy maybe effective in malignant diseases for which DNA tumor viruses are etiologic agent
48 -angle glaucoma (POAG) is a degenerative eye disease for which early treatment is critical to mitigat
49 contribute to the development of Alzheimer's disease, for which early administration of NSAIDs is pro
50                             One of the major diseases for which early diagnosis is crucial is represe
51 rhosis (PBC) is a classical autoimmune liver disease for which effective immunomodulatory therapy is
52  hold promise for the treatment of high-risk disease for which effective therapies are currently lack
53 oendocrine differentiation (NED) is a lethal disease for which effective therapies are urgently neede
54 singly threatened by outbreaks of infectious disease for which effective vaccines exist.
55 omes, but also for treating common metabolic diseases for which effective therapies are currently lac
56 ochondrial disorders are devastating genetic diseases for which efficacious therapies are still an un
57 ches to harness T cells in inflammatory skin diseases for which efficacy next to unwanted side effect
58 d to the fragile X syndrome and Huntington's disease, for which enough quantitative data have been co
59  not just to blood cancers but to autoimmune diseases for which ex vivo CAR-T cell therapies have sho
60 a brucei causes African sleeping sickness, a disease for which existing chemotherapies are limited by
61 erosis is an inflammatory, neurodegenerative disease for which experimental autoimmune encephalomyeli
62                               JMML remains a disease for which few curative therapies are available o
63 work suggests new treatment strategies for a disease for which few therapeutic options exist.
64 ly viewed as a late complication of systemic disease, for which few effective treatment options exist
65  be informative for applications beyond rare diseases for which few samples exist with high-dimension
66 but heterogeneous subset of congenital heart disease for which gene identification has been difficult
67             In contrast to many other common diseases for which genetic association-studies have succ
68  are three examples of long-known infectious diseases for which global vaccination has dramatically r
69 enesis of a broad range of neurodegenerative diseases, for which glymphatic failure may constitute a
70          This type of model is applicable to diseases for which habitat suitability varies by climate
71 e cancer is one of the most common malignant diseases for which health-care intervention is sought wo
72            Glaucoma is a chronic progressive disease for which ideal treatment would provide a locali
73 eatment of psoriasis, and other inflammatory diseases for which IL-17 antagonists are currently teste
74  Malignant mesothelioma remains an incurable disease for which immune-modulatory therapies, such as e
75 clerosis (ALS) is a progressive motor neuron disease for which important subtypes are caused by varia
76 serous ovarian carcinoma (HGSOC) is a lethal disease for which improved screening and treatment strat
77 ensively reviewed, with a focus on prevalent diseases for which improved diagnostic techniques are in
78 's usage is limited by the specific task and disease for which it is trained.
79 4-dihydropyridines hold promise for the many diseases for which K(Ca)3.1 has been experimentally conf
80 e room for improvement exists with organisms/diseases for which laboratory methods are limited.
81 on, autism, and dystonia represent disabling diseases for which little etiologic information is avail
82 osis, or ATTRv amyloidosis, is a progressive disease, for which liver transplantation (LT) has been a
83 approaches could be applied to other chronic diseases for which long-term controlled trials are not f
84 n is a hallmark feature of peripheral artery disease for which minimal therapeutic options exist.
85 ng framework can readily be applied to other diseases for which mitochondrial dysfunction has been im
86         Pancreatic cancer is a highly lethal disease, for which mortality closely parallels incidence
87 d to data for cystic fibrosis and Huntington disease, for which mutations in disease genes have alrea
88 leukemia (CLL) is a clinically heterogeneous disease for which natural history can be predicted based
89 oma (ATC) is a highly aggressive form of the disease for which new therapeutic options are desperatel
90 c leukemia (CLL) is an incurable progressive disease for which new therapies are required.
91                                 Sarcoma is a disease for which new treatments are needed.
92                              End-stage liver disease for which no cause can be identified, cryptogeni
93 isease (COPD) is a prevalent smoking-related disease for which no disease-altering therapies currentl
94 steoarthritis is a common degenerative joint disease for which no disease-modifying drugs are current
95  (HDV) is a cause of acute and chronic liver disease for which no effective therapy is currently avai
96     Idiopathic pulmonary fibrosis is a fatal disease for which no effective treatment exists.
97 iabetes is a serious, genetically influenced disease for which no fully effective treatments are avai
98         Osteoarthritis (OA) is a nonsystemic disease for which no oral or parenteral disease-modifyin
99 threatening, tick-borne, emerging infectious disease for which no satisfactory animal model has been
100   C3 glomerulopathy (C3G) is a severe kidney disease for which no specific therapy exists.
101 spillover to human populations, cause severe disease for which no specific treatment is available.
102 tients at an academic uveitis practice had a disease for which no SUN classification criteria existed
103 lpha2 chain), is a severe and incapacitating disease for which no therapy is yet available.
104  complication of the scleroderma spectrum of disease for which no treatment has been proven effective
105 mary pulmonary hypertension is a progressive disease for which no treatment has been shown in a prosp
106             Pneumonic plague, an often-fatal disease for which no vaccine is presently available, res
107 stis causes pneumonic plague, an often-fatal disease for which no vaccine is presently available.
108 e for tackling the transmission of dengue, a disease for which no vaccine, prophylaxis, or therapeuta
109                                         Lyme disease, for which no human vaccine is currently availab
110 on specific medicines to treat human genetic diseases for which no cures currently exist.
111 r myopathies (CNM) are non-dystrophic muscle diseases for which no effective therapy is currently ava
112  cGAS by self-DNA leads to severe autoimmune diseases for which no effective treatment is available y
113 opathies (TSEs) are fatal, neurodegenerative diseases for which no effective treatments are available
114 ed for treating a wide range of neurological diseases for which no effective treatments are available
115 ishmaniases are globally important parasitic diseases for which no human vaccines are currently avail
116 oup of fatal transmissible neurodegenerative diseases for which no pharmacological treatment is avail
117 ical potential in response to new infectious diseases for which no specific drugs or vaccines are ava
118 tauopathies are still severe and untreatable diseases for which no validated biomarkers exist.
119  cause acute, often fatal, hemorrhagic fever diseases, for which no effective vaccines are currently
120  Metastatic colorectal cancer is a prevalent disease for which novel targeted therapies and biologica
121 lmonary fibrosis (IPF) is a progressive lung disease for which novel therapies are needed.
122           Glioblastoma remains a devastating disease for which novel therapies are urgently needed.
123  there are many gastrointestinal and hepatic diseases for which obesity is the direct cause (eg, nona
124 ) is a genetically heterogeneous and complex disease for which only one locus, APOE, has been definit
125 tion of admixture mapping in complex genetic diseases for which only modest ethnicity risk ratios of
126  nearsightedness, is a common ocular genetic disease for which over 20 candidate genomic loci have be
127  of prevention strategies aiming at emerging diseases for which parameters are not nessecerly known i
128 ution of their genomic variation to rare eye diseases, for which pathogenic non-coding variants are e
129 spective, we review several of the lysosomal diseases for which PCs have been studied and the SAR of
130 s assessing current health status or risk of disease, for which personal risk may be reduced by modif
131 frequent and heterogeneous inflammatory skin disease, for which personalized medicine remains a chall
132 tery disease is a potentially incapacitating disease for which pharmacological options are limited.
133 PSC) is an immune-mediated cholestatic liver disease for which pharmacological treatment options are
134 s (AAAs) are a chronic inflammatory vascular disease for which pharmacological treatments are not ava
135 mpsia are similar to those in cardiovascular disease, for which physical activity is shown to be prot
136 l disease (IBD) is a complex multi-factorial disease for which physiologically relevant in vitro mode
137 te or land cover, and for direct transmitted diseases for which population density and mitigation pra
138 y (HCM) is an important genetic heart muscle disease for which prevalence in the general population h
139 family that is responsible for a fatal human disease for which preventative or therapeutic measures d
140 onable genotypes in 73 genes associated with diseases for which preventive or therapeutic measures ar
141 sus (SLE) is an uncommon, clinically complex disease for which prior experience treating similar pati
142 ectal cancer (CRC) is a highly heterogeneous disease, for which prognosis has been relegated to clini
143             Pertussis is a highly contagious disease for which prompt, point-of-care diagnosis remain
144 t for tuberculosis, malaria, AIDS, and other diseases for which protective vaccines do not exist.
145 vere epilepsies and other neurodevelopmental diseases for which rare, often de novo, mutations play a
146 or investigating optimal treatments for rare diseases for which RCTs are not always feasible, identif
147 e recommendations for 13 vaccine-preventable diseases for which recommendations were in place prior t
148  prevalent, debilitating, and costly chronic disease for which recommended first-line treatment is un
149 tudies of vector-borne disease, such as Lyme disease, for which risk is intrinsically linked to geogr
150  of chronic diseases, such as coronary heart disease, for which rural areas now exceed urban ones.
151                                         With diseases for which screening to exclude disease represen
152 a distinctive phenotype of neurodegenerative disease for which several causative genes have been iden
153 es mellitus and neurodegeneration are common diseases for which shared genetic factors are still only
154   We have made every attempt to review those diseases for which significant and confirmatory evidence
155 tory, except for diabetes and chronic kidney disease, for which smaller, but significant, risk reduct
156 mited because these models focus narrowly on diseases for which some drugs already exist.
157 -1 infection, but also numerous inflammatory diseases for which such antiplatelet therapies are curre
158 ease (PD) is a progressive neurodegenerative disease for which susceptibility is linked to genetic an
159      Otitis media (OM) is a common pediatric disease for which systemic antibiotics are often prescri
160 s leishmaniasis is an ultimately self-curing disease for which systemic therapy with pentavalent anti
161 fy APLAID as a G-CSF-driven autoinflammatory disease, for which targeted therapy is feasible.
162 e that is enriched for patients with genetic diseases for which targeted therapies are available.
163 um enzyme normalized in 97% of patients with diseases for which testings exist.
164 g of this prediagnostic phase of Parkinson's disease (for which the clinical diagnostic and prognosti
165                         As the first genetic disease for which the culpable gene was identified by po
166  nephritis is a potentially fatal autoimmune disease for which the current treatment is ineffective a
167                    Chronic pancreatitis is a disease for which the diagnosis may be difficult to asce
168 with a recessive form of Charcot-Marie-Tooth disease for which the genetic basis had not been identif
169 (FMD) is a rare, nonatherosclerotic arterial disease for which the molecular basis is unknown.
170 Plp1, as illustrated by Pelizaeus-Merzbacher disease for which the most common cause is PLP1 overexpr
171 ymphocyte Hodgkin lymphoma (NLPHL) is a rare disease for which the optimal therapy is unknown.
172 sis (ALS) is a devastating neurodegenerative disease for which the origin and underlying cellular def
173 pathic pulmonary fibrosis (IPF) is a complex disease for which the pathogenesis is poorly understood.
174 generative disease with a focus on Alzheimer disease, for which the evidence is most compelling.
175 lied to prioritize genes for rare and orphan diseases for which the molecular basis is unknown.
176 predispose certain individuals to infectious diseases for which the sensing of microbial cell compone
177  apply to a number of more common autoimmune diseases for which the underlying genetics and mechanism
178 identified numerous loci linked with complex diseases, for which the molecular mechanisms remain larg
179  for diseases such as the inflammatory bowel diseases, for which the pathogenesis is unknown and an e
180 individuals can develop often fatal invasive disease for which therapeutic options are limited.
181 on of AAV technologies to a wider variety of diseases for which therapeutic transgenes exceed the pac
182        Melioidosis is an emerging infectious disease for which there are currently no licensed vaccin
183 topenia syndrome (SFTS) is an emerging viral disease for which there are currently no therapeutic opt
184                         PPH is a progressive disease for which there are few effective therapies.
185       Sarcoidosis is a chronic granulomatous disease for which there are limited therapeutic options.
186    Osteoarthritis (OA) is a widespread joint disease for which there are no disease-modifying treatme
187 ) is the most severe form of alcoholic liver disease for which there are no effective therapies.
188 e therapeutic benefit against an intractable disease for which there are no effective treatments and
189 myelitis (AFM) is a devastating neurological disease for which there are no treatments of proven effi
190 atients with advanced hereditary MTC, a rare disease for which there has been no effective therapy.
191 rtic aneurysms (AAAs) are a life-threatening disease for which there is a lack of effective therapy p
192 ypertension (PAH) is a progressive and fatal disease for which there is an ever-expanding body of gen
193               Inhalation anthrax is a deadly disease for which there is currently no effective treatm
194 ally for late-stage and androgen-independent disease for which there is currently no effective treatm
195 dystrophy (DMD) is a devastating progressive disease for which there is currently no effective treatm
196 frequent and life-threatening cardiovascular disease for which there is currently no medical treatmen
197   Refractory focal epilepsy is a devastating disease for which there is frequently no effective treat
198 s (NASH) is a leading cause of chronic liver disease for which there is limited therapy available.
199         Sepsis is a common, life-threatening disease for which there is little treatment.
200  cilia cause PCD, a debilitating respiratory disease for which there is no approved therapy.
201 se is a progressive, fatal neurodegenerative disease for which there is no cure.
202 lerosis (ALS) is a progressive neuromuscular disease for which there is no cure.
203                  Food allergy is a prevalent disease for which there is no current treatment beyond c
204 ataxia, is a polyglutamine neurodegenerative disease for which there is no disease-modifying therapy.
205  Fibrosis is a serious complication of Crohn disease for which there is no effective therapy.
206 ocytic leukemia (JMML) is an early childhood disease for which there is no effective therapy.
207  drugs for treatment of infected hosts for a disease for which there is no evidence of acquired immun
208 tohepatitis (NASH) is a common chronic liver disease for which there is no known effective therapy.
209             Aortic stenosis is a progressive disease for which there is no known medical treatment to
210 MPV) is a leading cause of acute respiratory disease for which there is no licensed vaccine.
211 nopathy (PVR) is a recurring and problematic disease for which there is no pharmacologic treatment.
212 s an inherited progressive neurodegenerative disease for which there is no proven disease-modifying t
213 heimer's disease is a common and devastating disease for which there is no readily available biomarke
214  a highly contagious, frequently fatal human disease for which there is no specific antiviral treatme
215 is a heterogeneous and clinically aggressive disease for which there is no targeted therapy.
216 MD) is a progressive X-linked muscle wasting disease for which there is no treatment.
217 iva (HS) is a chronic debilitating cutaneous disease for which there is no universally effective trea
218 patients warrants further investigation in a disease for which there is no US Food and Drug Administr
219  colitis is an important worldwide parasitic disease for which there is not a well-established animal
220            Multiple sclerosis, an autoimmune disease for which there is still no cure, is characteriz
221   K-Ras-induced lung cancer is a very common disease, for which there are currently no effective ther
222 red for research and educational use in this disease, for which there is currently no approved treatm
223 is (ALS) is a devastating fatal motor neuron disease, for which there is currently no cure or effecti
224  be developed as a new therapy for Stargardt disease, for which there is currently no treatment.
225 ase is an important cause of end-stage renal disease, for which there is no proven therapy.
226  the pathogenesis of a range of inflammatory diseases for which there are currently no effective ther
227          iPS cells can also be used to study diseases for which there are no adequate human in vitro
228  capable of causing severe hemorrhagic fever diseases for which there are no approved vaccines and li
229 mentia (ALS/FTD) are major neurodegenerative diseases for which there are no cures.
230  Inflammation drives pathology in many human diseases for which there are no disease-modifying drugs.
231     Scleroderma is a group of skin-fibrosing diseases for which there are no effective treatments.
232            However, there remain a number of diseases for which there are no or only partially effect
233 osis represents one of the largest groups of diseases for which there are very few effective therapie
234                                          For diseases for which there could be maternal effects on ri
235  whooping cough, unlike many other childhood diseases for which there is an efficacious vaccine, has
236 eted therapies for a group of chronic kidney diseases for which there is currently a great unmet need
237 y efficacious in the treatment of autoimmune diseases for which there is no association with rs180069
238 (IBDs) are a set of complex and debilitating diseases for which there is no satisfactory treatment.
239 irus genus, whose members cause severe human diseases for which there is no specific treatment.
240 llnesses such as Alzheimer's and Parkinson's diseases, for which there are no cures.
241 e in neuroinflammatory and neurodegenerative diseases, for which there is an unmet therapeutic need.
242 helium (RPE) 65 are associated with blinding diseases, for which there is no effective therapy allevi
243  enhanced the incidence of neurodegenerative diseases, for which there is, as yet, no cure.
244 d effective vaccines against both infectious diseases--for which there are currently no effective lic
245  for finding drugs that can be used to treat diseases for which they are currently not prescribed.
246 yrins, and they are implicated in many human diseases, for which they serve as biomarkers and therape
247 fication policies aimed at the prevention of diseases for which this genotype is associated with incr
248 vaccines need to be developed against deadly diseases for which this strategy is often not feasible b
249 onditions as fatigue in chronic inflammatory diseases, for which to date there are no specific treatm
250 is (PSC) is a rare, but serious, cholestatic disease for which, to date, no effective therapy exists
251 her chronic diseases, such as end-stage lung disease, for which transplant may be the only effective
252 arkers in osteosarcoma, a rare, recalcitrant disease for which treatment standards have not changed i
253 phoblastic leukemia (ALL) is a heterogeneous disease, for which treatment guidelines are still evolvi
254 ntrast with the dopaminergic symptoms of the disease, for which treatment is available, non-motor sym
255 able parasitaemia, in contrast to many other diseases for which treatment (and/or diagnosis) may be b
256 ended duration were common for the principal diseases for which treatment was initiated, with 27.3% o
257 r of a novel class of disparate degenerative diseases for which UPR activation and signaling is the c
258 oid cancer, and especially intermediate-risk disease, for which use of adjuvant radioactive iodine an
259 eventable blindness and sexually transmitted diseases for which vaccines are badly needed.
260 her medically important sexually transmitted diseases for which vaccines are sought but which have pr
261  address the prevention of slowly developing diseases for which validated biomarkers are rare.
262  drugs and drug targets for the treatment of diseases for which validated therapeutic pathways are no
263 er neurologic, hematologic, and inflammatory diseases for which viral etiologies have been suggested.
264 ion for iron in the pathogenesis of this new disease, for which we propose the name 'neuroferritinopa
265 nable engineering of resistance in plants to diseases for which we currently lack robust genetic resi
266                              We separate the diseases for which we have individual genotype data into
267 is are among the priority neglected zoonotic diseases for which WHO advocates control.

 
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